EA201690312A1 - Улучшенная липидная композиция - Google Patents

Улучшенная липидная композиция

Info

Publication number
EA201690312A1
EA201690312A1 EA201690312A EA201690312A EA201690312A1 EA 201690312 A1 EA201690312 A1 EA 201690312A1 EA 201690312 A EA201690312 A EA 201690312A EA 201690312 A EA201690312 A EA 201690312A EA 201690312 A1 EA201690312 A1 EA 201690312A1
Authority
EA
Eurasian Patent Office
Prior art keywords
lipid composition
improved lipid
lipid
formula
improved
Prior art date
Application number
EA201690312A
Other languages
English (en)
Other versions
EA028860B1 (ru
Inventor
Акин Акинг
Джозеф Р. Доркин
Сяоцзюнь Цинь
Вильям Кентли
Мутиах Манохаран
Каллантоттатил Г. Раджив
Джаяпракаш К. Нараянаннаир
Мутусами Джаяраман
Цзяньсинь Чэнь
Стивен Энселл
Original Assignee
Текмира Фармасьютикалз Корпорэйшн
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Текмира Фармасьютикалз Корпорэйшн filed Critical Текмира Фармасьютикалз Корпорэйшн
Publication of EA201690312A1 publication Critical patent/EA201690312A1/ru
Publication of EA028860B1 publication Critical patent/EA028860B1/ru

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • A61K47/551—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1277—Processes for preparing; Proliposomes
    • A61K9/1278—Post-loading, e.g. by ion or pH gradient
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C227/00—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C227/14—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton from compounds containing already amino and carboxyl groups or derivatives thereof
    • C07C227/18—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton from compounds containing already amino and carboxyl groups or derivatives thereof by reactions involving amino or carboxyl groups, e.g. hydrolysis of esters or amides, by formation of halides, salts or esters
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C227/00—Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C227/38—Separation; Purification; Stabilisation; Use of additives
    • C07C227/40—Separation; Purification
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C29/00—Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom not belonging to a six-membered aromatic ring
    • C07C29/58—Preparation of compounds having hydroxy or O-metal groups bound to a carbon atom not belonging to a six-membered aromatic ring by elimination of halogen, e.g. by hydrogenolysis, splitting-off
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D475/00—Heterocyclic compounds containing pteridine ring systems
    • C07D475/02—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4
    • C07D475/04—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Emergency Medicine (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)

Abstract

Изобретение относится к катионному липиду формулы (I), улучшенной липидной композиции, содержащей катионный липид формулы I, и соответствующим способам применения. Также описаны нацеливающие липиды и специфичные липидные композиции, содержащие указанные нацеливающие липиды.
EA201690312A 2009-06-10 2010-06-10 Улучшенная липидная композиция EA028860B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18580009P 2009-06-10 2009-06-10
US24483409P 2009-09-22 2009-09-22

Publications (2)

Publication Number Publication Date
EA201690312A1 true EA201690312A1 (ru) 2016-08-31
EA028860B1 EA028860B1 (ru) 2018-01-31

Family

ID=43309230

Family Applications (3)

Application Number Title Priority Date Filing Date
EA201190306A EA024960B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201791744A EA201791744A3 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201690312A EA028860B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция

Family Applications Before (2)

Application Number Title Priority Date Filing Date
EA201190306A EA024960B1 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция
EA201791744A EA201791744A3 (ru) 2009-06-10 2010-06-10 Улучшенная липидная композиция

Country Status (24)

Country Link
US (4) US8158601B2 (ru)
EP (2) EP3431076B1 (ru)
JP (4) JP5819291B2 (ru)
KR (7) KR102066189B1 (ru)
CN (2) CN104873464B (ru)
AU (4) AU2010259984B2 (ru)
CA (2) CA2764609C (ru)
CY (2) CY1120641T1 (ru)
DK (2) DK2440183T3 (ru)
EA (3) EA024960B1 (ru)
ES (2) ES2901627T3 (ru)
HK (1) HK1212620A1 (ru)
HR (2) HRP20211619T1 (ru)
HU (2) HUE038796T2 (ru)
IL (4) IL216876A (ru)
LT (2) LT2440183T (ru)
MX (4) MX2011013320A (ru)
NZ (3) NZ596958A (ru)
PL (2) PL3431076T3 (ru)
PT (2) PT2440183T (ru)
SG (3) SG176786A1 (ru)
SI (2) SI2440183T1 (ru)
TR (1) TR201811076T4 (ru)
WO (1) WO2010144740A1 (ru)

Families Citing this family (665)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
EP2584048B1 (en) 2006-05-11 2014-07-23 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
MX2010008394A (es) * 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
JP2011518117A (ja) * 2008-03-05 2011-06-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｅｇ５およびＶＥＧＦ遺伝子の発現を阻害するための組成物および方法
WO2009127060A1 (en) 2008-04-15 2009-10-22 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
JP5529142B2 (ja) 2008-09-25 2014-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 血清アミロイドａ遺伝子の発現を阻害するための脂質製剤組成物および方法
DK2937418T3 (en) 2008-10-20 2018-01-22 Alnylam Pharmaceuticals Inc Compositions and Methods for Inhibiting Transthyretin Expression
CN105709229B (zh) 2008-11-10 2020-07-28 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
US8324368B2 (en) 2008-12-10 2012-12-04 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
US20100267806A1 (en) * 2009-03-12 2010-10-21 David Bumcrot LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES
WO2010129687A1 (en) * 2009-05-05 2010-11-11 Alnylam Pharmaceuticals, Inc Methods of delivering oligonucleotides to immune cells
EP3431076B1 (en) * 2009-06-10 2021-10-06 Arbutus Biopharma Corporation Improved lipid formulation
US8273869B2 (en) * 2009-06-15 2012-09-25 Alnylam Pharmaceuticals, Inc. Lipid formulated dsRNA targeting the PCSK9 gene
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
EP2810643A3 (en) 2009-08-14 2015-03-11 Alnylam Pharmaceuticals Inc. Lipid formulated compositions and mehods for inhibiting expression of a gene from the ebola virus
CN102639115A (zh) 2009-09-15 2012-08-15 阿尔尼拉姆医药品有限公司 脂质配制的组合物及抑制eg5和vegf基因的表达的方法
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
PL2480658T3 (pl) 2009-09-22 2017-12-29 Medicago Inc. Sposób otrzymywania VLP pochodzenia roślinnego
WO2011056883A1 (en) 2009-11-03 2011-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (ttr)
US20110244026A1 (en) 2009-12-01 2011-10-06 Braydon Charles Guild Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
US20130017223A1 (en) * 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
WO2011088309A1 (en) 2010-01-14 2011-07-21 Regulus Therapeutics Inc. Microrna compositions and methods
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
JP2013527856A (ja) 2010-05-12 2013-07-04 プロチバ バイオセラピューティクス インコーポレイティッド 陽イオン性脂質およびその使用方法
WO2012000104A1 (en) * 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
BR112013000244A2 (pt) 2010-07-06 2016-05-17 Novartis Ag lipossomas com lipídeos apresentando pka vantajoso para administração de rna
DK2591114T3 (en) 2010-07-06 2016-08-29 Glaxosmithkline Biologicals Sa Immunization of large mammals with low doses of RNA
WO2012016188A2 (en) * 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2807307C (en) 2010-08-17 2021-02-09 Merck Sharp & Dohme Corp. Rna interference mediated inhibition of hepatitis b virus (hbv) gene expression using short interfering nucleic acid (sina)
EP2609106A4 (en) 2010-08-26 2014-03-19 Merck Sharp & Dohme RNA INTERFERENCE-MEDIATED INHIBITION OF EXPRESSION OF PHD2 GENE (PROLYL HYDROXYLASE DOMAIN 2) USING SMALL INTERFERING NUCLEIC ACID (PANI)
DK4066857T3 (da) 2010-08-31 2023-02-20 Glaxosmithkline Biologicals Sa Pegylerede liposomer til afgivelse af immunogen-kodende RNA
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
EP3943114A1 (en) 2010-09-20 2022-01-26 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
LT3590949T (lt) 2010-10-01 2022-07-25 Modernatx, Inc. Ribonukleorūgštys, kurių sudėtyje yra n1-metil-pseudouracilų, ir jų naudojimas
CA2814386C (en) 2010-10-11 2019-08-20 Novartis Ag Antigen delivery platforms
EP3485913A1 (en) 2010-10-21 2019-05-22 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
EP2635265B1 (en) 2010-11-05 2018-04-04 Sirna Therapeutics, Inc. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
TWI620816B (zh) 2011-03-23 2018-04-11 苜蓿股份有限公司 植物衍生蛋白回收方法
JP2014511687A (ja) 2011-03-31 2014-05-19 モデルナ セラピューティクス インコーポレイテッド 工学操作された核酸の送達および製剤
WO2012151575A2 (en) * 2011-05-05 2012-11-08 Duke University A method of controlling coagulation
AU2012260434A1 (en) * 2011-05-23 2013-10-31 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
EP3388068A1 (en) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Composition and methods for inhibition of expression of protein c (proc) genes
EP2723758B1 (en) 2011-06-21 2018-06-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof
KR20230084331A (ko) * 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
CA2840989A1 (en) 2011-07-06 2013-01-10 Novartis Ag Immunogenic combination compositions and uses thereof
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
MX354267B (es) 2011-10-03 2018-02-21 Moderna Therapeutics Inc Star Nucléosidos, nucleótidos, y ácidos nucleicos modificados, y usos de los mismos.
SG11201402392QA (en) 2011-11-18 2014-06-27 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
WO2013086322A1 (en) * 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
CA2856742A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
US20130156849A1 (en) * 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
RU2718053C2 (ru) 2012-02-24 2020-03-30 Протива Байотерапьютикс Инк. Триалкиловые катионные липиды и способы их использования
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US10501513B2 (en) * 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
JP2015518705A (ja) 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CA2868996A1 (en) * 2012-04-02 2013-10-10 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CA2870941C (en) 2012-04-19 2021-05-25 Sirna Therapeutics, Inc. Diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
WO2013170146A1 (en) 2012-05-10 2013-11-14 Uab Research Foundation Methods and compositions for modulating mir-204 activity
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
US20140120157A1 (en) * 2012-09-19 2014-05-01 Georgetown University Targeted liposomes
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
MX367076B (es) 2012-12-05 2019-08-05 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina convertasa subtilisina kexina 9 (pcsk9) y metodos de uso de las mismas.
WO2014089239A1 (en) * 2012-12-07 2014-06-12 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
EP2946014A2 (en) 2013-01-17 2015-11-25 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
CN104254320A (zh) * 2013-03-06 2014-12-31 百奥迈科生物技术有限公司 脂质体制剂、制备方法及其应用
US20160024181A1 (en) * 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
CN105209490A (zh) 2013-03-14 2015-12-30 夏尔人类遗传性治疗公司 用于递送mrna编码的抗体的方法和组合物
IL290953B2 (en) 2013-03-14 2024-01-01 Ethris Gmbh CFTR mRNA Assemblies and Related Methods and Uses
EP3467108B1 (en) 2013-03-14 2024-05-22 Translate Bio, Inc. Methods for purification of messenger rna
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
ES2649490T3 (es) 2013-03-14 2018-01-12 Alnylam Pharmaceuticals, Inc. Composiciones de ARNi contra el componente C5 del complemento y métodos para su uso
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
CN105143456A (zh) * 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
EP2968147A4 (en) * 2013-03-15 2017-02-22 The Penn State Research Foundation Acid stable liposomal compositions
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
ES2795249T3 (es) 2013-03-15 2020-11-23 Translate Bio Inc Mejora sinérgica de la administración de ácidos nucleicos a través de formulaciones mezcladas
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
RU2019110030A (ru) 2013-05-01 2019-05-06 Ионис Фармасьютикалз, Инк. Композиции и способы
EP2994167B1 (en) 2013-05-06 2020-05-06 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
BR112015029276B1 (pt) 2013-05-22 2022-07-12 Alnylam Pharmaceuticals, Inc Agente de irna fita dupla capaz de inibir a expressão de tmprss6, composição farmacêutica e uso dos mesmos
SG10201804472YA (en) 2013-05-22 2018-07-30 Alnylam Pharmaceuticals Inc SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2015006747A2 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use.
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US10195291B2 (en) * 2013-09-24 2019-02-05 Alnylam Pharmaceuticals, Inc. Compositions and methods for the manufacture of lipid nanoparticles
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
CN105658800A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 Mrna的cns递送及其用途
US9522176B2 (en) 2013-10-22 2016-12-20 Shire Human Genetic Therapies, Inc. MRNA therapy for phenylketonuria
KR102096796B1 (ko) 2013-10-22 2020-05-27 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna의 전달을 위한 지질 제형
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
CN105873902B (zh) 2013-11-18 2019-03-08 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
KR20230037686A (ko) 2013-11-22 2023-03-16 미나 테라퓨틱스 리미티드 Ｃ/ｅｂｐ 알파 짧은 활성화 ｒｎａ 조성물 및 사용 방법
IL282401B (en) 2013-12-12 2022-08-01 Alnylam Pharmaceuticals Inc Complementary component irna compositions and methods for using them
EP3093283B1 (en) 2014-01-09 2018-12-19 Eisai R&D Management Co., Ltd. Cationic lipid
JP6594902B2 (ja) 2014-02-11 2019-10-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物及びその使用方法
EP3556353A3 (en) 2014-02-25 2020-03-18 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
EA201691696A1 (ru) 2014-04-25 2017-03-31 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
US9926556B2 (en) 2014-04-28 2018-03-27 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
DK3137596T3 (da) 2014-05-01 2019-09-02 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression
DK3137605T3 (da) 2014-05-01 2020-12-14 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af angiopoietin-lignende-3-ekspression
AU2015252917B2 (en) 2014-05-01 2019-09-26 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating PKK expression
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
MX2016014306A (es) 2014-05-02 2017-06-12 Cerenis Therapeutics Holding Sa Marcadores para terapia con lipoproteinas de alta densidad (hdl).
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
BR112016026950B1 (pt) 2014-05-22 2023-03-07 Alnylam Pharmaceuticals, Inc Agente de ácido ribonucleico (rnai) de fita dupla, seus usos e composição farmacêutica
CA2949106C (en) 2014-05-30 2023-10-24 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
US20170145424A1 (en) * 2014-06-06 2017-05-25 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
PE20171238A1 (es) 2014-06-24 2017-08-24 Shire Human Genetic Therapies Composiciones enriquecidas estereoquimicamente para administracion de acidos nucleicos
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA2953265C (en) 2014-07-02 2023-09-26 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
EP4159741A1 (en) 2014-07-16 2023-04-05 ModernaTX, Inc. Method for producing a chimeric polynucleotide encoding a polypeptide having a triazole-containing internucleotide linkage
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
ES2928500T3 (es) 2014-08-29 2022-11-18 Alnylam Pharmaceuticals Inc Patisirán para su uso en el tratamiento de amiloidosis mediada por transtiretina
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3209780A4 (en) 2014-10-24 2018-09-19 University of Maryland, Baltimore Short non-coding protein regulatory rnas (sprrnas) and methods of use
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
US9943595B2 (en) 2014-12-05 2018-04-17 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
JP2018504380A (ja) 2014-12-18 2018-02-15 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｒｅｖｅｒｓｉｒ（商標）化合物
JP6592458B2 (ja) 2014-12-26 2019-10-16 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
JP6895892B2 (ja) 2015-03-19 2021-06-30 トランスレイト バイオ， インコーポレイテッド ポンペ病のｍＲＮＡ治療
MX2017012610A (es) 2015-04-08 2018-03-16 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
IL295159A (en) 2015-04-13 2022-09-01 Alnylam Pharmaceuticals Inc Preparations of angiopoietin-like 3 irna and methods of using them
WO2016176426A1 (en) 2015-04-28 2016-11-03 Duke University Thrombus imaging aptamers and methods of using same
EP3292201A2 (en) 2015-05-06 2018-03-14 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
US10221127B2 (en) 2015-06-29 2019-03-05 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
PE20180800A1 (es) 2015-07-10 2018-05-09 Ionis Pharmaceuticals Inc Moduladores de diaciglicerol aciltransferasa 2 (dgat2)
AU2016303661B2 (en) 2015-07-31 2020-12-10 Arcturus Therapeutics, Inc. Multiligand agent for drug delivery
SG10201912341SA (en) 2015-07-31 2020-02-27 Alnylam Pharmaceuticals Inc TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
SG10202007937SA (en) 2015-09-02 2020-09-29 Alnylam Pharmaceuticals Inc PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2017048620A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
EP3350157B1 (en) 2015-09-17 2022-01-05 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2017053722A1 (en) 2015-09-24 2017-03-30 Ionis Pharmaceuticals, Inc. Modulators of kras expression
JP6893505B2 (ja) * 2015-10-02 2021-06-23 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドコンジュゲーション方法
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
CN114686548A (zh) 2015-10-14 2022-07-01 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
US20190046555A1 (en) 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
KR20180073606A (ko) 2015-11-06 2018-07-02 아이오니스 파마수티컬즈, 인코포레이티드 아포리포프로테인 (a) 발현 조정
ES2864102T3 (es) 2015-11-18 2021-10-13 Provivi Inc Microorganismos para la producción de feromonas de insectos y compuestos relacionados
IL259795B2 (en) 2015-12-07 2024-04-01 Genzyme Corp Methods and preparations for the treatment of diseases related to SERPINC1
EP3386484B1 (en) 2015-12-10 2022-03-30 ModernaTX, Inc. Compositions and methods for delivery of therapeutic agents
JP2018536689A (ja) 2015-12-10 2018-12-13 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ステロール調節エレメント結合タンパク質（ＳＲＥＢＰ）シャペロン（ＳＣＡＰ）ｉＲＮＡ組成物およびその使用方法
JP7114465B2 (ja) 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
ES2919552T3 (es) 2015-12-23 2022-07-27 Modernatx Inc Procedimientos de utilización de polinucleotidos codificadores de ligando ox40
WO2017120612A1 (en) 2016-01-10 2017-07-13 Modernatx, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
EP3405579A1 (en) 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
SG11201806663TA (en) 2016-03-03 2018-09-27 Univ Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
WO2017156012A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
WO2017180917A2 (en) 2016-04-13 2017-10-19 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
CN109152735B (zh) * 2016-05-09 2022-03-11 阿斯利康(瑞典)有限公司 包含亲脂性抗炎剂的脂质纳米颗粒及其使用方法
WO2017201340A2 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding relaxin
WO2017201328A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
US20190298657A1 (en) 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
DK3458083T3 (da) 2016-05-18 2023-01-30 Modernatx Inc Polynukleotider, der koder for interleukin-12 (il12), og anvendelser heraf
CA3024625A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding citrin for the treatment of citrullinemia type 2
EP3464311A4 (en) * 2016-06-06 2020-03-18 Provivi, Inc. HALF-BIOSYNTHETIC PRODUCTION OF FATTY ALCOHOLS AND FATTY ALDEHYDES
JP2019518028A (ja) 2016-06-10 2019-06-27 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 補体成分Ｃ５ｉＲＮＡ組成物及び発作性夜間血色素尿症（ＰＮＨ）を処置するためのその使用方法
EP3842530A1 (en) 2016-06-13 2021-06-30 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
EP3476832B1 (en) 2016-06-24 2023-04-19 Eisai R&D Management Co., Ltd. Cationic lipid
AU2017286980B2 (en) 2016-06-30 2023-10-26 Arbutus Biopharma Corporation Compositions and methods for delivering messenger RNA
PT3484524T (pt) 2016-07-15 2023-02-15 Ionis Pharmaceuticals Inc Composições e métodos para a modulação de smn2
GB201614120D0 (en) * 2016-08-18 2016-10-05 Univ Of Kwazulu-Natal pH-responsive lipids
WO2018033254A2 (en) 2016-08-19 2018-02-22 Curevac Ag Rna for cancer therapy
WO2018041921A1 (en) 2016-08-31 2018-03-08 Curevac Ag Mixing device for the production of a liquid nucleic acid composition
KR102493872B1 (ko) 2016-09-02 2023-01-30 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
UY37145A (es) 2016-09-02 2019-08-30 Arrowhead Pharmaceuticals Inc Ligandos de direccionamiento
DK3436054T4 (da) 2016-09-13 2022-08-29 Allergan Inc Stabiliserede ikke-protein clostridtoxinsammensætninger
EP3512526A4 (en) 2016-09-16 2020-05-27 Duke University ACTIVE SUBSTANCES AGAINST WILLEBRAND FACTOR (VWF) AND METHOD FOR USE THEREOF
EP3519578B1 (en) 2016-10-03 2021-12-22 Precision Nanosystems Inc Compositions for transfecting resistant cell types
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
KR20190086681A (ko) 2016-10-26 2019-07-23 모더나티엑스, 인크. 면역 반응을 향상시키기 위한 메신저 리보핵산 및 그의 사용 방법
WO2018089540A1 (en) 2016-11-08 2018-05-17 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
WO2018104538A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rna for treatment or prophylaxis of a liver disease
WO2018104540A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rnas for wound healing
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018115525A1 (en) 2016-12-23 2018-06-28 Curevac Ag Lassa virus vaccine
US11524066B2 (en) 2016-12-23 2022-12-13 CureVac SE Henipavirus vaccine
EP3558356A2 (en) 2016-12-23 2019-10-30 CureVac AG Mers coronavirus vaccine
US20190351039A1 (en) 2017-02-01 2019-11-21 Modernatx, Inc. Immunomodulatory therapeutic mrna compositions encoding activating oncogene mutation peptides
WO2018146506A1 (en) * 2017-02-10 2018-08-16 Universitat Politècnica De València Therapeutic derivatives
MA47603A (fr) 2017-02-27 2020-01-01 Translate Bio Inc Nouvel arnm cftr à codons optimisés
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
US11203569B2 (en) 2017-03-15 2021-12-21 Modernatx, Inc. Crystal forms of amino lipids
SG11201907916TA (en) 2017-03-15 2019-09-27 Modernatx Inc Compounds and compositions for intracellular delivery of therapeutic agents
JP2020514370A (ja) 2017-03-17 2020-05-21 キュアバック アーゲー 組合せ抗癌療法のためのｒｎａワクチン及び免疫チェックポイント阻害剤
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
KR20190133699A (ko) 2017-03-24 2019-12-03 큐어백 아게 Crispr-연관 단백질을 암호화하는 핵산 및 이의 용도
WO2018187590A1 (en) 2017-04-05 2018-10-11 Modernatx, Inc. Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
MA50278A (fr) 2017-04-18 2020-02-26 Alnylam Pharmaceuticals Inc Méthodes pour le traitement de sujets atteints d'une infection par le virus de l'hépatite b (vhb)
JP7297676B2 (ja) 2017-04-28 2023-06-26 アクイタス セラピューティクス インコーポレイテッド 新規なカルボニル脂質、および核酸を送達するための脂質ナノ粒子製剤
AU2018268859A1 (en) 2017-05-16 2019-12-12 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
US11104921B2 (en) 2017-05-17 2021-08-31 Provivi, Inc. Microorganisms for the production of insect pheromones and related compounds
US20230064106A1 (en) 2017-05-17 2023-03-02 Curevac Real Estate Gmbh Method for determining at least one quality parameter of an rna sample
CA3063723A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (il12) polypeptides and uses thereof
WO2018213789A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Modified messenger rna comprising functional rna elements
US11015204B2 (en) 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
WO2018222925A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for phenylketonuria
WO2018222926A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
WO2018226854A2 (en) 2017-06-06 2018-12-13 Wayne State University Antifouling polymer coatings and reverse coating method
CN109030429B (zh) * 2017-06-09 2022-06-21 南京大学 一种可对活细胞内microRNA21进行原位成像的纳米放大自参比探针
MA49395A (fr) 2017-06-14 2020-04-22 Modernatx Inc Polynucléotides codant pour le facteur viii de coagulation
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
EP3641834B1 (en) 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
CN111328287A (zh) 2017-07-04 2020-06-23 库瑞瓦格股份公司 新型核酸分子
JP7277432B2 (ja) 2017-07-13 2023-05-19 アルナイラム ファーマシューティカルズ， インコーポレイテッド 乳酸脱水素酵素Ａ（ＬＤＨＡ）ｉＲＮＡ組成物及びその使用方法
JP7355731B2 (ja) 2017-08-16 2023-10-03 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
WO2019036670A2 (en) 2017-08-18 2019-02-21 Modernatx, Inc. EFFECTIVE MRNA VACCINES
EP3673069A1 (en) 2017-08-22 2020-07-01 CureVac AG Bunyavirales vaccine
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019048631A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
BR112020005230A2 (pt) 2017-09-19 2020-09-24 Alnylam Pharmaceuticals, Inc. composições e métodos para o tratamento da amiloidose mediada por transtiretina (ttr)
MX2020003995A (es) 2017-10-19 2020-07-22 Curevac Ag Nuevas moleculas de acido nucleico artificiales.
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2019092153A1 (en) 2017-11-08 2019-05-16 Curevac Ag Rna sequence adaptation
US20210236644A1 (en) 2017-11-10 2021-08-05 Cocoon Biotech Inc. Ocular applications of silk-based products
WO2019099610A1 (en) 2017-11-16 2019-05-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019104198A2 (en) * 2017-11-22 2019-05-31 Beckman Coulter, Inc. Diluent preparation modules and units
EP3714047A2 (en) 2017-11-22 2020-09-30 ModernaTX, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
CA3079543A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
CA3079428A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11931406B2 (en) 2017-12-13 2024-03-19 CureVac SE Flavivirus vaccine
CA3086343A1 (en) 2017-12-18 2019-06-27 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
AU2018392716A1 (en) 2017-12-20 2020-06-18 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
JP7164547B2 (ja) 2017-12-27 2022-11-01 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
MA51523A (fr) 2018-01-05 2020-11-11 Modernatx Inc Polynucléotides codant pour des anticorps anti-virus du chikungunya
WO2019140452A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
CA3089117A1 (en) 2018-01-30 2019-08-08 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
EP3752612A4 (en) 2018-02-12 2021-11-10 Ionis Pharmaceuticals, Inc. MODIFIED COMPOUNDS AND USES THEREOF
US20210361761A1 (en) 2018-04-05 2021-11-25 Curevac Ag Novel yellow fever nucleic acid molecules for vaccination
AU2019251449A1 (en) * 2018-04-09 2020-11-12 Orgenesis Inc. Bioxomes particles, redoxomes, method and composition
WO2019200171A1 (en) 2018-04-11 2019-10-17 Modernatx, Inc. Messenger rna comprising functional rna elements
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
CN112292395A (zh) 2018-04-17 2021-01-29 库瑞瓦格股份公司 用于疫苗接种的新型rsv rna分子和组合物
JP7438135B2 (ja) 2018-05-09 2024-02-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｆｘｉの発現を低下させるための化合物及び方法
EP3790607B1 (en) 2018-05-11 2023-12-27 Lupagen, Inc. Systems for closed loop, real-time modifications of patient cells
TW202016304A (zh) 2018-05-14 2020-05-01 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
MA52709A (fr) 2018-05-23 2021-03-31 Modernatx Inc Administration d'adn
CN112437767B (zh) 2018-05-24 2023-10-27 川斯勒佰尔公司 硫酯阳离子脂质
JP7463006B2 (ja) 2018-05-30 2024-04-08 トランスレイト バイオ， インコーポレイテッド ステロイド性部分を含むカチオン性脂質
JP7441802B2 (ja) 2018-05-30 2024-03-01 トランスレイト バイオ， インコーポレイテッド ビタミンカチオン性脂質
JP2021525725A (ja) 2018-05-30 2021-09-27 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａワクチンおよびその使用
AU2019277355A1 (en) 2018-05-30 2020-12-17 Translate Bio, Inc. Phosphoester cationic lipids
KR102591743B1 (ko) 2018-06-08 2023-10-19 후지필름 가부시키가이샤 화합물 또는 그 염 및 지질 입자
WO2020002525A1 (en) 2018-06-27 2020-01-02 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
EP3813798A1 (en) 2018-06-28 2021-05-05 AstraZeneca AB Exosome extracellular vesicles and methods of use
SG11202100602SA (en) 2018-07-23 2021-02-25 Translate Bio Inc Dry power formulations for messenger rna
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020033748A1 (en) 2018-08-08 2020-02-13 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
WO2020033791A1 (en) 2018-08-09 2020-02-13 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
US20210170005A1 (en) 2018-08-15 2021-06-10 University Of Florida Research Foundation, Inc. Methods of sensitizing tumors to treatment with immune checkpoint inhibitors
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法
AU2019333042A1 (en) 2018-08-29 2021-03-04 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
EP3846776A1 (en) 2018-09-02 2021-07-14 ModernaTX, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
EP3849597A1 (en) 2018-09-12 2021-07-21 University of Florida Research Foundation, Inc. Slow-cycling cell-rna based nanoparticle vaccine to treat cancer
JP2022500436A (ja) 2018-09-13 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
US20220243182A1 (en) 2018-09-13 2022-08-04 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
EP3849617A1 (en) 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
MA53615A (fr) 2018-09-14 2021-07-21 Modernatx Inc Polynucléotides codant pour le polypeptide a1, de la famille de l'uridine diphosphate glycosyltransférase 1, pour le traitement du syndrome de crigler-najjar
CA3112781A1 (en) 2018-09-14 2020-03-19 Modernatx, Inc. Methods and compositions for treating cancer using mrna therapeutics
CN112424355A (zh) 2018-09-18 2021-02-26 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
MA53734A (fr) 2018-09-27 2021-08-04 Modernatx Inc Polynucléotides codant pour l'arginase 1 pour le traitement d'une déficience en arginase
ES2951836T3 (es) * 2018-10-01 2023-10-25 Alnylam Pharmaceuticals Inc Lípidos biodegradables para el suministro de agentes activos
KR20210093232A (ko) 2018-10-09 2021-07-27 더 유니버시티 오브 브리티시 콜롬비아 유기용매와 세제가 없는 형질감염 적격 소포를 포함하는 조성물과 시스템 및 관련 방법
WO2020081933A1 (en) 2018-10-19 2020-04-23 Translate Bio, Inc. Pumpless encapsulation of messenger rna
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
US20220001026A1 (en) 2018-11-08 2022-01-06 Modernatx, Inc. Use of mrna encoding ox40l to treat cancer in human patients
WO2020097511A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
CA3117882A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
EP3876994A2 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Peg lipidoid compounds
EP3877444A1 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. Multi-peg lipid compounds
WO2020102172A2 (en) 2018-11-12 2020-05-22 Translate Bio, Inc. Methods for inducing immune tolerance
EP3883917B1 (en) 2018-11-21 2024-01-24 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
CN113710799A (zh) 2018-11-28 2021-11-26 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
US20220040281A1 (en) 2018-12-21 2022-02-10 Curevac Ag Rna for malaria vaccines
CN113412273A (zh) 2019-01-07 2021-09-17 川斯勒佰尔公司 用于治疗原发性纤毛运动障碍的组合物和方法
CN113474328A (zh) 2019-01-11 2021-10-01 爱康泰生治疗公司 用于脂质纳米颗粒递送活性剂的脂质
WO2020160430A1 (en) 2019-01-31 2020-08-06 Modernatx, Inc. Vortex mixers and associated methods, systems, and apparatuses thereof
EP3917503B1 (en) 2019-01-31 2024-05-08 ModernaTX, Inc. Methods of preparing lipid nanoparticles
US20220133908A1 (en) 2019-02-08 2022-05-05 Curevac Ag Coding rna administered into the suprachoroidal space in the treatment of ophthalmic diseases
CN113924365A (zh) 2019-03-29 2022-01-11 迪克纳制药公司 用于治疗kras相关疾病或病症的组合物和方法
US20220204994A1 (en) 2019-04-05 2022-06-30 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
WO2020208361A1 (en) 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
US20220233444A1 (en) 2019-04-22 2022-07-28 Translate Bio, Inc. Thioester cationic lipids
WO2020242720A1 (en) 2019-05-02 2020-12-03 University Of Florida Research Foundation, Inc. Compositions for treatment of diffuse intrinsic pontine glioma
JP2022530678A (ja) 2019-05-03 2022-06-30 ディセルナ ファーマシューティカルズ インコーポレイテッド 短縮センス鎖を有する二本鎖核酸阻害剤分子
US20220257724A1 (en) 2019-05-03 2022-08-18 Translate Bio, Inc. Di-thioester cationic lipids
WO2020227537A1 (en) 2019-05-07 2020-11-12 Modernatx, Inc Differentially expressed immune cell micrornas for regulation of protein expression
US20230085318A1 (en) 2019-05-07 2023-03-16 Modernatx, Inc. Polynucleotides for disrupting immune cell activity and methods of use thereof
EP3965806A1 (en) 2019-05-08 2022-03-16 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
JP2022532078A (ja) 2019-05-08 2022-07-13 アストラゼネカ アクチボラグ 皮膚及び創傷のための組成物並びにその使用の方法
AU2020280105A1 (en) 2019-05-22 2022-01-20 Massachusetts Institute Of Technology Circular RNA compositions and methods
EP3976593A1 (en) 2019-05-31 2022-04-06 Translate Bio, Inc. Macrocyclic lipids
AU2020288624A1 (en) 2019-06-04 2022-02-03 Cocoon Biotech Inc. Silk-based products, formulations, and methods of use
JP7241869B2 (ja) * 2019-06-07 2023-03-17 富士フイルム株式会社 脂質組成物
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
WO2020257716A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Tricine and citric acid lipids
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
WO2020263883A1 (en) 2019-06-24 2020-12-30 Modernatx, Inc. Endonuclease-resistant messenger rna and uses thereof
WO2020263985A1 (en) 2019-06-24 2020-12-30 Modernatx, Inc. Messenger rna comprising functional rna elements and uses thereof
CN114390921A (zh) 2019-07-08 2022-04-22 川斯勒佰尔公司 改善的负载mrna的脂质纳米颗粒和其制备方法
WO2021009336A1 (en) 2019-07-18 2021-01-21 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for inducing full ablation of hematopoiesis
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
AU2020315962A1 (en) 2019-07-23 2022-02-03 Translate Bio, Inc. Stable compositions of mRNA-loaded lipid nanoparticles and processes of making
US20220280639A1 (en) 2019-07-31 2022-09-08 Modernatx, Inc. Compositions and methods for delivery of rna interference agents to immune cells
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
WO2021026358A1 (en) 2019-08-07 2021-02-11 Moderna TX, Inc. Compositions and methods for enhanced delivery of agents
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
JOP20220037A1 (ar) 2019-08-14 2023-01-30 Acuitas Therapeutics Inc جسيمات نانوية دهنية محسنة لتوصيل الأحماض النووية
JP2022544412A (ja) 2019-08-14 2022-10-18 キュアバック アーゲー 免疫賦活特性が減少したｒｎａ組み合わせおよび組成物
KR20220047826A (ko) 2019-08-16 2022-04-19 리제너론 파마슈티칼스 인코포레이티드 고 농도의 항-c5 제형
WO2021046265A1 (en) 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
EP4031524A1 (en) 2019-09-19 2022-07-27 ModernaTX, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
CN114585738A (zh) * 2019-09-20 2022-06-03 中美瑞康核酸技术(南通)研究院有限公司 用于治疗血小板减少症的核酸分子及其应用
JP2022548320A (ja) 2019-09-23 2022-11-17 オメガ セラピューティクス， インコーポレイテッド アポリポタンパク質ｂ（ａｐｏｂ）遺伝子発現をモジュレートするための組成物および方法
CA3147643A1 (en) 2019-09-23 2021-04-01 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (hnf4.alpha.) gene expression
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
CA3158013A1 (en) 2019-10-15 2021-04-22 Modernatx, Inc. Mrnas encoding granulocyte-macrophage colony stimulating factor for treating parkinson's disease
WO2021076828A1 (en) 2019-10-18 2021-04-22 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
WO2021081058A1 (en) 2019-10-21 2021-04-29 Translate Bio, Inc. Compositions, methods and uses of messenger rna
AU2020369515A1 (en) 2019-10-22 2022-04-21 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
CN111205994B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株具核梭杆菌动物亚种菌株及其应用
CN111004738B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株具核梭杆菌多形亚种分离株及其应用
CN110903998B (zh) * 2019-10-23 2021-09-21 上海市第十人民医院 一株肠道分离的具核梭杆菌文氏亚种菌株及其应用
CN115052889A (zh) 2019-10-25 2022-09-13 瑞泽恩制药公司 用于治疗或预防c5相关疾病的给药方案
MX2022004388A (es) 2019-11-01 2022-05-06 Alnylam Pharmaceuticals Inc Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
TW202132568A (zh) 2019-11-13 2021-09-01 美商阿尼拉製藥公司 用於治療血管收縮素原相關病症之方法及組成物
CN117417265A (zh) 2019-11-15 2024-01-19 富士胶片株式会社 脂质组合物
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
JPWO2021117770A1 (ru) * 2019-12-10 2021-06-17
JPWO2021117769A1 (ru) * 2019-12-10 2021-06-17
WO2021119226A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
CA3165388A1 (en) 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
KR20220142432A (ko) 2019-12-20 2022-10-21 트랜슬레이트 바이오 인코포레이티드 메신저 rna의 직장 전달
IL293890A (en) 2019-12-20 2022-08-01 Curevac Ag Lipid nanoparticles for delivery of nucleic acids
CN110974954B (zh) * 2019-12-24 2021-03-16 珠海丽凡达生物技术有限公司 一种用于增强核酸疫苗免疫效果的脂质纳米颗粒及其制备方法
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
WO2021142280A1 (en) 2020-01-10 2021-07-15 Modernatx, Inc. Methods of making tolerogenic dendritic cells
IL294599A (en) 2020-01-15 2022-09-01 Dicerna Pharmaceuticals Inc Methylene phosphonate nucleic acids and their analogs
KR20220133957A (ko) 2020-01-31 2022-10-05 모더나티엑스, 인크. 지질 나노입자의 제조 방법
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2021156267A1 (en) 2020-02-04 2021-08-12 Curevac Ag Coronavirus vaccine
IL295445A (en) 2020-02-10 2022-10-01 Alnylam Pharmaceuticals Inc Preparations and methods for silencing vegf-a expression
BR112022016324A2 (pt) 2020-02-18 2022-10-11 Alnylam Pharmaceuticals Inc Composições de irna de apolipoproteína c3 (apoc3) e métodos de uso das mesmas
WO2021173840A1 (en) 2020-02-25 2021-09-02 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
PE20221913A1 (es) 2020-02-28 2022-12-23 Ionis Pharmaceuticals Inc Compuestos y metodos para modular smn2
CN115484932A (zh) * 2020-03-04 2022-12-16 维乎医疗有限公司 用于靶向rna递送的组合物和方法
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
IL296109A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Ketohexokinase (khk) IRNA compositions and methods of using them
CA3171231A1 (en) 2020-03-09 2021-09-16 Sean Michael Sullivan Compositions and methods for inducing immune responses
EP4118207A1 (en) 2020-03-11 2023-01-18 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
AU2021244559A1 (en) 2020-03-24 2022-11-17 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing Gaucher therapeutics
CN116209759A (zh) 2020-03-26 2023-06-02 阿尔尼拉姆医药品有限公司 冠状病毒iRNA组合物及其使用方法
WO2021202772A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Multilamellar rna nanoparticle vaccine against sars-cov-2
CN117377763A (zh) 2020-04-06 2024-01-09 阿尔尼拉姆医药品有限公司 用于沉默myoc表达的组合物和方法
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
AU2021251754A1 (en) 2020-04-07 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing SCN9A expression
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
MX2022013525A (es) 2020-04-27 2023-01-24 Alnylam Pharmaceuticals Inc Composiciones de agentes de ácido ribonucleico de interferencia (arni) de apolipoproteína e (apoe) y métodos de uso de las mismas.
BR112022021136A2 (pt) 2020-04-30 2022-11-29 Alnylam Pharmaceuticals Inc Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
KR20230008801A (ko) 2020-05-07 2023-01-16 트랜슬레이트 바이오 인코포레이티드 Sars-cov-2 항원을 암호화하는 최적화된 뉴클레오티드 서열
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
US20230181619A1 (en) 2020-05-07 2023-06-15 Translate Bio, Inc. Improved compositions for cftr mrna therapy
JP2023526059A (ja) 2020-05-14 2023-06-20 モダーナティエックス・インコーポレイテッド ｍＲＮＡ治療薬及びエフェクター分子を含むＬＮＰ組成物
US20230226219A1 (en) 2020-05-14 2023-07-20 Translate Bio, Inc. Peg lipidoid compounds
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
EP4149425A1 (en) 2020-05-15 2023-03-22 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021234548A1 (en) 2020-05-18 2021-11-25 Max Biology Co. Ltd. Lipid-polymer compositions and methods of use
JP2023526422A (ja) 2020-05-20 2023-06-21 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー コロナウイルス抗原組成物及びそれらの使用
JP2023526423A (ja) 2020-05-20 2023-06-21 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 免疫原性組成物及びその使用
WO2021243207A1 (en) 2020-05-28 2021-12-02 Modernatx, Inc. Use of mrnas encoding ox40l, il-23 and il-36gamma for treating cancer
CA3180101A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
BR112022024248A2 (pt) 2020-05-29 2023-10-10 CureVac SE Vacinas de combinação à base de ácido nucleico
US20230203509A1 (en) 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
US20230233475A1 (en) 2020-06-01 2023-07-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
AU2021285812A1 (en) 2020-06-01 2023-01-05 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
TW202214856A (zh) 2020-06-18 2022-04-16 美商阿尼拉製藥公司 黃嘌呤脫氫酶（ＸＤＨ）ｉＲＮＡ組成物及其使用之方法
JP2023531511A (ja) 2020-06-23 2023-07-24 モダーナティエックス・インコーポレイテッド 半減期が延長されたｍＲＮＡ治療薬を含むＬＮＰ組成物
WO2022006527A1 (en) 2020-07-02 2022-01-06 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
EP4178613A1 (en) 2020-07-08 2023-05-17 Janssen Sciences Ireland Unlimited Company Rna replicon vaccines against hbv
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
US11976019B2 (en) 2020-07-16 2024-05-07 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
KR20230040353A (ko) 2020-07-17 2023-03-22 그린라이트 바이오사이언시스, 아이엔씨. 유전 장애를 위한 핵산 치료제
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
CA3170741A1 (en) 2020-07-31 2022-02-03 Curevac Ag Nucleic acid encoded antibody mixtures
EP4192505A1 (en) 2020-08-04 2023-06-14 Dicerna Pharmaceuticals, Inc. Systemic delivery of oligonucleotides
US20230277457A1 (en) 2020-08-06 2023-09-07 Modernatx, Inc. Methods of preparing lipid nanoparticles
US20240066114A1 (en) 2020-08-31 2024-02-29 CureVac SE Multivalent nucleic acid based coronavirus vaccines
JP2023542492A (ja) 2020-09-03 2023-10-10 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 免疫原性組成物及びその使用
CN116056728A (zh) 2020-09-14 2023-05-02 富士胶片株式会社 脂质组合物
IL301540A (en) 2020-09-23 2023-05-01 Translate Bio Inc TES-based cationic lipids
WO2022066916A1 (en) 2020-09-23 2022-03-31 Translate Bio, Inc. Piperazine-based cationic lipids
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
CN116615233A (zh) * 2020-09-25 2023-08-18 Dnarx公司 用于使生物分子在受试者中表达的***和方法
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
CA3194409A1 (en) 2020-10-05 2022-04-14 Sunil Shah Cannabinoid-containing compositions and use for treating and preventing diseases
CA3194863A1 (en) 2020-10-06 2022-04-14 Shrirang KARVE Improved process and formulation of lipid nanoparticles
US20230365995A1 (en) 2020-10-07 2023-11-16 Precision Biosciences, Inc. Lipid nanoparticle compositions
WO2022081548A1 (en) 2020-10-12 2022-04-21 Translate Bio, Inc. Improved process of preparing ice-based lipid nanoparticles
AU2021361986A1 (en) 2020-10-12 2023-06-15 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
WO2022087041A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
JP2023548587A (ja) 2020-11-09 2023-11-17 トランスレイト バイオ， インコーポレイテッド コドン最適化したｍＲＮＡの送達のための改善された組成物
IL302625A (en) 2020-11-13 2023-07-01 Modernatx Inc Cystic fibrosis-encoding polynucleotides transmembrane conductance regulator for the treatment of cystic fibrosis
AU2021380809A1 (en) 2020-11-13 2023-06-22 Alnylam Pharmaceuticals, Inc. COAGULATION FACTOR V (F5) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022109139A1 (en) 2020-11-18 2022-05-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
MX2023006133A (es) 2020-11-25 2023-07-18 Translate Bio Inc Formulaciones estables de nanopartículas de lipídos líquidos.
WO2022112855A1 (en) 2020-11-27 2022-06-02 Guangzhou Ribobio Co., Ltd Lipid compound and the composition thereof
BR112021021361A2 (pt) 2020-11-27 2023-10-10 Guangzhou Ribobio Co Ltd Composto lipídico e composição do mesmo
WO2022110099A1 (en) 2020-11-30 2022-06-02 Guangzhou Argorna Biopharmaceuticals Co., Ltd. Coronavirus vaccines and uses thereof
TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制ｈａｏ１（羥基酸氧化酶１（乙醇酸氧化酶））基因表現的方法及組成物
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CA3171051A1 (en) 2020-12-22 2022-06-30 Curevac Ag Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
WO2022137133A1 (en) 2020-12-22 2022-06-30 Curevac Ag Rna vaccine against sars-cov-2 variants
WO2022140702A1 (en) 2020-12-23 2022-06-30 Flagship Pioneering, Inc. Compositions of modified trems and uses thereof
EP4267740A1 (en) 2020-12-28 2023-11-01 Arcturus Therapeutics, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
CN116981692A (zh) 2021-01-14 2023-10-31 翻译生物公司 递送mRNA编码的抗体的方法和组合物
WO2022162027A2 (en) 2021-01-27 2022-08-04 Curevac Ag Method of reducing the immunostimulatory properties of in vitro transcribed rna
EP4289814A1 (en) 2021-02-04 2023-12-13 Shionogi & Co., Ltd Cationic lipid
CA3206911A1 (en) 2021-02-08 2022-08-11 Sang M. Lee Unsaturated dendrimers compositions,related formulations, and methods of use thereof
JP2024506908A (ja) 2021-02-12 2024-02-15 モデルナティエックス インコーポレイテッド インビボ療法のためのペイロードを含むｌｎｐ組成物
JP2024508714A (ja) 2021-02-12 2024-02-28 アルナイラム ファーマシューティカルズ， インコーポレイテッド スーパーオキシドジスムターゼ１－（ＳＯＤ１－）関連神経変性疾患を治療または予防するためのスーパーオキシドジスムターゼ１（ＳＯＤ１）ｉＲＮＡ組成物およびその使用方法
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
BR112023016903A2 (pt) 2021-02-26 2023-10-10 Ethris Gmbh Formulação de suspensão aquosa para formação de aerossol, nebulizador, e, aerossol
EP4298218A1 (en) 2021-02-26 2024-01-03 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
AU2022231003A1 (en) 2021-03-04 2023-09-14 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
WO2022204370A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2022204549A1 (en) 2021-03-25 2022-09-29 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
CA3212653A1 (en) 2021-03-26 2022-09-29 Glaxosmithkline Biologicals Sa Immunogenic compositions
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
AU2022246895A1 (en) 2021-03-31 2023-10-19 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
CA3171429A1 (en) 2021-03-31 2022-09-30 Alexander SCHWENGER Syringes containing pharmaceutical compositions comprising rna
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
EP4314332A2 (en) 2021-04-01 2024-02-07 ModernaTX, Inc. Methods for identification and ratio determination of rna species in multivalent rna compositions
TW202309002A (zh) 2021-04-15 2023-03-01 美商轉譯生技公司 基於「古德」緩衝液的陽離子脂質
CA3215606A1 (en) 2021-04-19 2022-10-27 Shrirang KARVE Improved compositions for delivery of mrna
CA3214538A1 (en) 2021-04-20 2022-10-27 Joel DE BEER Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
WO2022226318A1 (en) 2021-04-23 2022-10-27 Modernatx, Inc. Isoquinoline-stabilized lipid nanoparticle formulations
US20220370616A1 (en) 2021-04-23 2022-11-24 Modernatx, Inc. Stabilized formulations
BR112023022284A2 (pt) 2021-04-26 2023-12-26 Alnylam Pharmaceuticals Inc Composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma
IL308404A (en) 2021-04-27 2024-01-01 Generation Bio Co Non-viral DNA vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4329731A1 (en) 2021-04-29 2024-03-06 ModernaTX, Inc. Lyophilization methods for preparing lipid formulated therapeutics
EP4334446A1 (en) 2021-05-03 2024-03-13 CureVac SE Improved nucleic acid sequence for cell type specific expression
EP4337177A1 (en) 2021-05-11 2024-03-20 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
WO2022246020A1 (en) 2021-05-19 2022-11-24 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
EP4341405A1 (en) 2021-05-20 2024-03-27 Korro Bio, Inc. Methods and compositions for adar-mediated editing
CN113546180A (zh) * 2021-05-25 2021-10-26 重庆医科大学 一种具有心肌靶向性的基因递送载体及其制备方法
AU2022282812A1 (en) * 2021-05-28 2023-11-09 Nanovation Therapeutics Inc. Mc3-type lipids and use thereof in the preparation of lipid nanoparticles
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
IL308743A (en) 2021-06-04 2024-01-01 Alnylam Pharmaceuticals Inc Compositions of human chromosome 9 open reading frame 72 iRNA factor (C9ORF72) and methods of using them
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
WO2022260772A1 (en) * 2021-06-09 2022-12-15 Carnegie Mellon University Lipid nanoparticle formulations for gastrointestinal delivery
WO2022266083A2 (en) 2021-06-15 2022-12-22 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
KR102516680B1 (ko) * 2021-06-24 2023-04-03 주식회사 테르나테라퓨틱스 지질 나노 입자 및 그 제조방법
WO2022270941A1 (ko) * 2021-06-24 2022-12-29 주식회사 테르나테라퓨틱스 지질 나노 입자 및 그 제조방법
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
EP4363580A1 (en) 2021-06-30 2024-05-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
CN117881395A (zh) 2021-07-01 2024-04-12 翻译生物公司 用于递送mRNA的组合物
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023287751A1 (en) 2021-07-12 2023-01-19 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
CA3226887A1 (en) 2021-07-21 2023-01-26 Alnylam Pharmaceuticals, Inc. Metabolic disorder-associated target gene irna compositions and methods of use thereof
WO2023003995A1 (en) 2021-07-23 2023-01-26 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
WO2023009499A1 (en) 2021-07-27 2023-02-02 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease type 1a (gsd1a)
CA3227440A1 (en) 2021-07-27 2023-02-02 Stand Therapeutics Co., Ltd. Peptide tag and nucleic acid encoding same
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
EP4377460A1 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377331A2 (en) 2021-07-30 2024-06-05 CureVac SE Mrnas for treatment or prophylaxis of liver diseases
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023014649A1 (en) 2021-08-02 2023-02-09 Modernatx, Inc. Extraction-less reverse phase (rp) chromatography of mrna encapsulated in lipid nanoparticles for mrna purity assessment
AU2022323090A1 (en) 2021-08-03 2024-02-01 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
AU2022324003A1 (en) 2021-08-04 2024-02-08 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
WO2023015261A1 (en) 2021-08-04 2023-02-09 Modernatx, Inc. Mrnas encoding chimeric metabolic reprogramming polypeptides and uses thereof
WO2023014974A1 (en) 2021-08-06 2023-02-09 University Of Iowa Research Foundation Double stranded mrna vaccines
WO2023018773A1 (en) 2021-08-11 2023-02-16 Modernatx, Inc. Lipid nanoparticle formulations and methods of synthesis thereof
WO2023019181A1 (en) 2021-08-11 2023-02-16 Modernatx, Inc. Sars-cov-2 lipid nanoparticle vaccine formulations
CA3228733A1 (en) 2021-08-13 2023-02-16 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
AU2022337090A1 (en) 2021-09-03 2024-02-15 Glaxosmithkline Biologicals Sa Substitution of nucleotide bases in self-amplifying messenger ribonucleic acids
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
CA3230056A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids comprising phosphatidylserine
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CA3232635A1 (en) 2021-09-17 2023-03-23 Flagship Pioneering Innovations Vi, Llc Compositions and methods for producing circular polyribonucleotides
WO2023044094A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
WO2023056401A1 (en) 2021-10-01 2023-04-06 Modernatx, Inc. Rna formulations for high volume distribution, and methods of using the same for treating a disease or condition caused by or associated with human cytomegalovirus
WO2023056044A1 (en) 2021-10-01 2023-04-06 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
WO2023064469A1 (en) 2021-10-13 2023-04-20 Modernatx, Inc. Compositions of mrna-encoded il15 fusion proteins and methods of use thereof
WO2023064530A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
WO2023069895A1 (en) 2021-10-18 2023-04-27 Modernatx, Inc. Markerless dna production
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
CA3235362A1 (en) 2021-10-26 2023-05-04 Astrazeneca Ab Novel lipids for delivery of nucleic acid segments
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023077170A1 (en) 2021-11-01 2023-05-04 Modernatx, Inc. Polynucleotides encoding integrin beta-6 and methods of use thereof
CA3236235A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
WO2023086893A1 (en) 2021-11-10 2023-05-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
KR20230068047A (ko) 2021-11-10 2023-05-17 주식회사 에스엠엘바이오팜 트레할로즈 유도체 및 신규 구조 유지 화합물을 포함하는 핵산 의약품 전달용 지질나노입자의 약학 조성물
WO2023092060A1 (en) 2021-11-18 2023-05-25 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
CA3237573A1 (en) 2021-11-18 2023-05-25 Astrazeneca Ab Novel lipids for delivery of nucleic acid segments
WO2023092151A1 (en) * 2021-11-22 2023-05-25 Ohio State Innovation Foundation Compositions and methods for the treatment of neurodegenerative disorders
WO2023096990A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovation Vi, Llc Coronavirus immunogen compositions and their uses
WO2023097003A2 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and their uses
AU2022397292A1 (en) 2021-11-24 2024-05-30 Flagship Pioneering Innovations Vi, Llc Varicella-zoster virus immunogen compositions and their uses
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
WO2023107999A2 (en) 2021-12-08 2023-06-15 Modernatx, Inc. Herpes simplex virus mrna vaccines
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023114307A1 (en) 2021-12-15 2023-06-22 Modernatx, Inc. Determination of encapsulation efficiency of lipid nanoparticles
WO2023114889A1 (en) 2021-12-16 2023-06-22 Modernatx, Inc. Processes for preparing lipid nanoparticles
TW202340461A (zh) 2021-12-22 2023-10-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物和方法
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
TW202342064A (zh) 2021-12-23 2023-11-01 美商旗艦先鋒創新有限責任公司 編碼抗融合多肽之環狀多核糖核苷酸
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
CN114957027B (zh) 2022-01-13 2023-05-19 北京悦康科创医药科技股份有限公司 一种阳离子脂质化合物、包含其的组合物及用途
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023135298A1 (en) 2022-01-17 2023-07-20 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of inducing cell death of a population of solid tumor cells
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
WO2023147090A1 (en) 2022-01-27 2023-08-03 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023152365A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of the 15-lipoxygenase for the treatment of lymphedema
WO2023159197A1 (en) 2022-02-18 2023-08-24 Modernatx, Inc. Mrnas encoding checkpoint cancer vaccines and uses thereof
WO2023161350A1 (en) 2022-02-24 2023-08-31 Io Biotech Aps Nucleotide delivery of cancer therapy
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023177655A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
WO2023178167A1 (en) 2022-03-16 2023-09-21 Translate Bio, Inc. Asymmetric piperazine-based cationic lipids
WO2023177904A1 (en) 2022-03-18 2023-09-21 Modernatx, Inc. Sterile filtration of lipid nanoparticles and filtration analysis thereof for biological applications
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023193002A1 (en) 2022-04-01 2023-10-05 Modernatx, Inc. Cross mixers for lipid nanoparticle production, and methods of operating the same
WO2023196399A1 (en) 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023196988A1 (en) 2022-04-07 2023-10-12 Modernatx, Inc. Methods of use of mrnas encoding il-12
WO2023196634A2 (en) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
WO2023200893A1 (en) * 2022-04-13 2023-10-19 Oregon State University Small molecule enhancers of antisense oligo activity
WO2023198857A1 (en) 2022-04-13 2023-10-19 Sanofi "good" buffer-based cationic lipids
WO2023201296A1 (en) 2022-04-15 2023-10-19 Modernatx, Inc. Ribosomal engagement potency assay
WO2023212696A1 (en) 2022-04-29 2023-11-02 Modernatx, Inc. Lyophilized human cytomegalovirus vaccines
WO2023215498A2 (en) 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
WO2023220083A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
WO2023220734A2 (en) * 2022-05-13 2023-11-16 The Trustees Of The University Of Pennsylvania Bisphosphonate lipids, lipid nanoparticle compositions comprising the same, and methods of use thereof for targeted delivery
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
WO2023220729A2 (en) 2022-05-13 2023-11-16 Flagship Pioneering Innovations Vii, Llc Double stranded dna compositions and related methods
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2023230601A1 (en) 2022-05-27 2023-11-30 Beam Therapeutics Inc. Identification of nanoparticles for preferential tissue or cell targeting
WO2023239756A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
WO2023240277A2 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
CN117263818A (zh) * 2022-06-14 2023-12-22 杭州高田生物医药有限公司 阳离子脂质化合物及其制备方法和应用
WO2023242817A2 (en) 2022-06-18 2023-12-21 Glaxosmithkline Biologicals Sa Recombinant rna molecules comprising untranslated regions or segments encoding spike protein from the omicron strain of severe acute respiratory coronavirus-2
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
WO2024007020A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
WO2024015890A1 (en) 2022-07-13 2024-01-18 Modernatx, Inc. Norovirus mrna vaccines
WO2024023034A1 (en) 2022-07-25 2024-02-01 Institut National de la Santé et de la Recherche Médicale Use of apelin for the treatment of lymphedema
WO2024026254A1 (en) 2022-07-26 2024-02-01 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
WO2024026482A1 (en) 2022-07-29 2024-02-01 Modernatx, Inc. Lipid nanoparticle compositions comprising surface lipid derivatives and related uses
WO2024026487A1 (en) 2022-07-29 2024-02-01 Modernatx, Inc. Lipid nanoparticle compositions comprising phospholipid derivatives and related uses
WO2024026475A1 (en) 2022-07-29 2024-02-01 Modernatx, Inc. Compositions for delivery to hematopoietic stem and progenitor cells (hspcs) and related uses
US20240042021A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
WO2024030369A1 (en) 2022-08-01 2024-02-08 Modernatx, Inc. Extraction-less reverse phase (rp) chromatography for mrna purity assessment
WO2024035952A1 (en) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
US20240067968A1 (en) 2022-08-19 2024-02-29 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
WO2024044147A1 (en) 2022-08-23 2024-02-29 Modernatx, Inc. Methods for purification of ionizable lipids
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
WO2024042236A1 (en) 2022-08-26 2024-02-29 Ethris Gmbh Stable lipid or lipidoid nanoparticle suspensions
WO2024050483A1 (en) 2022-08-31 2024-03-07 Modernatx, Inc. Variant strain-based coronavirus vaccines and uses thereof
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024064642A2 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024064931A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of liver stage antigens and related methods
WO2024064934A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
WO2024068545A1 (en) 2022-09-26 2024-04-04 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
WO2024091918A2 (en) 2022-10-25 2024-05-02 Modernatx, Inc. Methods of lipid nanoparticle production in cross-mixers
US20240156949A1 (en) 2022-10-28 2024-05-16 Glaxosmithkline Biologicals Sa Nucleic Acid Based Vaccine
WO2024097639A1 (en) 2022-10-31 2024-05-10 Modernatx, Inc. Hsa-binding antibodies and binding proteins and uses thereof
WO2024097664A1 (en) 2022-10-31 2024-05-10 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
WO2024097874A1 (en) 2022-11-03 2024-05-10 Modernatx, Inc. Chemical stability of mrna
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
WO2024102799A1 (en) 2022-11-08 2024-05-16 Flagship Pioneering Innovations Vi, Llc Compositions and methods for producing circular polyribonucleotides
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions

Family Cites Families (239)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US105A (en) 1836-12-15 knight
US5218A (en) 1847-08-07 Improvement in plows
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (ru) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
US5008050A (en) 1984-06-20 1991-04-16 The Liposome Company, Inc. Extrusion technique for producing unilamellar vesicles
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
AU587989B2 (en) 1984-10-16 1989-09-07 Mitsubishi Chemical Corporation DMA fragments, expression vectors, proteins, hosts, and process for production of the proteins
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
AU580145B2 (en) 1985-02-13 1989-01-05 Scios Nova Inc. Human metallothionein-ii promoter in mammalian expression system
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
DE3687030T2 (de) 1985-03-15 1993-03-11 James Summerton Stereoregulare polynukleotiden bindende polymere.
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
EP0239631A4 (en) 1985-10-04 1989-01-12 Biotech Res Partners Ltd RECOMBINANT APOLIPOPROTEINS AND METHODS.
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
GB8712540D0 (en) 1987-05-28 1987-07-01 Ucb Sa Expression of human proapolipoprotein a-i
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
WO1988010264A1 (en) 1987-06-24 1988-12-29 Howard Florey Institute Of Experimental Physiology Nucleoside derivatives
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
WO1989009221A1 (en) 1988-03-25 1989-10-05 University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5149782A (en) 1988-08-19 1992-09-22 Tanox Biosystems, Inc. Molecular conjugates containing cell membrane-blending agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US4927637A (en) 1989-01-17 1990-05-22 Liposome Technology, Inc. Liposome extrusion method
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5473039A (en) 1989-08-18 1995-12-05 The Scripps Research Institute Polypeptide analogs of apolipoprotein E, diagnostic systems and methods using the analogs
US5168045A (en) 1989-08-18 1992-12-01 The Scripps Research Institute Diagnostic systems and methods using polypeptide analogs of apolipoprotein e
US5177189A (en) 1989-08-18 1993-01-05 The Scripps Research Institute Polypeptide analogs of Apolipoprotein E
US5182364A (en) 1990-02-26 1993-01-26 The Scripps Research Institute Polypeptide analogs of apolipoprotein E
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
EP0451221B1 (en) 1989-08-31 1994-10-12 City Of Hope Chimeric dna-rna catalytic sequences
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
EP0497875B1 (en) 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6153737A (en) 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
WO1992002258A1 (en) 1990-07-27 1992-02-20 Isis Pharmaceuticals, Inc. Nuclease resistant, pyrimidine modified oligonucleotides that detect and modulate gene expression
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
ATE131827T1 (de) 1990-08-03 1996-01-15 Sterling Winthrop Inc Verbindungen und verfahren zur unterdrückung der genexpression
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
CA2092002A1 (en) 1990-09-20 1992-03-21 Mark Matteucci Modified internucleoside linkages
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
PL169576B1 (pl) 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
DE69132510T2 (de) 1990-11-08 2001-05-03 Hybridon Inc Verbindung von mehrfachreportergruppen auf synthetischen oligonukleotiden
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
WO1993000443A1 (en) 1991-06-26 1993-01-07 Bio-Technology General Corp. Purification of recombinant apolipoprotein e from bacteria
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
SE9103701D0 (sv) 1991-12-13 1991-12-13 Kabi Pharmacia Ab Apolipoprotein
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US6372886B1 (en) 1992-06-23 2002-04-16 Arch Development Corp. Expression and purification of kringle domains of human apolipoprotein (a) in E. coli
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
EP0654077A4 (en) 1992-07-17 1996-03-13 Ribozyme Pharm Inc PROCESS AND REAGENT FOR THE TREATMENT OF DISEASES IN ANIMALS.
SE9203753D0 (sv) 1992-12-11 1992-12-11 Kabi Pharmacia Ab Expression system for producing apolipoprotein ai-m
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
WO1994022864A1 (en) 1993-03-30 1994-10-13 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
WO1994022891A1 (en) 1993-03-31 1994-10-13 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5532130A (en) 1993-07-20 1996-07-02 Dyad Pharmaceutical Corporation Methods and compositions for sequence-specific hybridization of RNA by 2'-5' oligonucleotides
ATE247128T1 (de) 1993-09-03 2003-08-15 Isis Pharmaceuticals Inc Aminoderivatisierte nukleoside und oligonukleoside
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5539083A (en) 1994-02-23 1996-07-23 Isis Pharmaceuticals, Inc. Peptide nucleic acid combinatorial libraries and improved methods of synthesis
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
DE69533569T2 (de) 1994-03-07 2006-01-05 Dendritic Nanotechnologies, Inc., Mt. Pleasant Bioaktive und/oder gezielte dendrimere-konjugate enthaltend genetisches material
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5840688A (en) 1994-03-22 1998-11-24 Research Corporation Technologies, Inc. Eating suppressant peptides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US6027726A (en) 1994-09-30 2000-02-22 Inex Phamaceuticals Corp. Glycosylated protein-liposome conjugates and methods for their preparation
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
WO1996011266A2 (en) 1994-10-05 1996-04-18 Amgen Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
SE9500778D0 (sv) 1995-03-03 1995-03-03 Pharmacia Ab Process for producing a protein
US5801155A (en) 1995-04-03 1998-09-01 Epoch Pharmaceuticals, Inc. Covalently linked oligonucleotide minor grove binder conjugates
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
IL122290A0 (en) 1995-06-07 1998-04-05 Inex Pharmaceuticals Corp Lipid-nucleic acid complex its preparation and use
US5672662A (en) 1995-07-07 1997-09-30 Shearwater Polymers, Inc. Poly(ethylene glycol) and related polymers monosubstituted with propionic or butanoic acids and functional derivatives thereof for biotechnical applications
US5672685A (en) 1995-10-04 1997-09-30 Duke University Source of apolipoprotein E and method of isolating apolipoprotein E
US6444806B1 (en) 1996-04-30 2002-09-03 Hisamitsu Pharmaceutical Co., Inc. Conjugates and methods of forming conjugates of oligonucleotides and carbohydrates
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
US6004925A (en) 1997-09-29 1999-12-21 J. L. Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6046166A (en) 1997-09-29 2000-04-04 Jean-Louis Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6037323A (en) 1997-09-29 2000-03-14 Jean-Louis Dasseux Apolipoprotein A-I agonists and their use to treat dyslipidemic disorders
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6335437B1 (en) 1998-09-07 2002-01-01 Isis Pharmaceuticals, Inc. Methods for the preparation of conjugated oligomers
US6534018B1 (en) 1998-11-13 2003-03-18 Optime Therapeutics, Inc. Method and apparatus for liposome production
WO2000030444A1 (en) * 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7390780B2 (en) * 1999-04-23 2008-06-24 Alza Corporation Gene delivery mediated by liposome-DNA complex with cleavable peg surface modification
EP1212085B1 (en) 1999-08-27 2007-10-31 INEX Pharmaceuticals Corp. Compositions for stimulating cytokine secretion and inducing an immune response
US6395437B1 (en) 1999-10-29 2002-05-28 Advanced Micro Devices, Inc. Junction profiling using a scanning voltage micrograph
EP1261620A2 (en) * 2000-02-07 2002-12-04 Roche Diagnostics Corporation Cationic amphiphiles for use in nucleic acid transfection
DK2796553T3 (da) 2000-03-30 2019-09-30 Whitehead Inst Biomedical Res Rna-sekvensspecifikke formidlere af rna-interferens
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
DK1407044T4 (en) 2000-12-01 2017-12-04 Max-Planck-Gesellschaft Zur Förderung Der Wss E V RNA interference mediating small RNA molecules
ES2307568T3 (es) 2000-12-08 2008-12-01 Coley Pharmaceutical Gmbh Acidos nucleicos de tipo cpg y metodos de uso de los mismos.
US20040009216A1 (en) * 2002-04-05 2004-01-15 Rodrigueza Wendi V. Compositions and methods for dosing liposomes of certain sizes to treat or prevent disease
US7887431B2 (en) 2008-05-16 2011-02-15 Taylor Made Golf Company, Inc. Golf club
ATE537263T1 (de) * 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Kationische lipide und verwendungsverfahren
CA2616877C (en) 2005-07-27 2014-01-28 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
CA2663601C (en) 2006-09-22 2014-11-25 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by rna interference
EP2068886B1 (en) 2006-10-03 2013-09-18 Tekmira Pharmaceuticals Corporation Lipid containing formulations
WO2009086558A1 (en) * 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
WO2009132131A1 (en) * 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
DK2937418T3 (en) * 2008-10-20 2018-01-22 Alnylam Pharmaceuticals Inc Compositions and Methods for Inhibiting Transthyretin Expression
CN105709229B (zh) 2008-11-10 2020-07-28 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
US20120149875A1 (en) * 2009-01-12 2012-06-14 Ge Healthcare Bio-Sciences Ab Affinity chromatography matrix
WO2010129687A1 (en) * 2009-05-05 2010-11-11 Alnylam Pharmaceuticals, Inc Methods of delivering oligonucleotides to immune cells
EP3431076B1 (en) 2009-06-10 2021-10-06 Arbutus Biopharma Corporation Improved lipid formulation
DE102009039097B3 (de) 2009-08-27 2010-11-25 Siemens Aktiengesellschaft Verfahren zum Übertragen von Daten in einem Sensornetzwerk, Sensorknoten und Zentral-Rechner
WO2011141704A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2012005769A1 (en) 2010-07-09 2012-01-12 Telecommunication Systems, Inc. Location privacy selector
US10867398B2 (en) 2017-11-21 2020-12-15 Reliance Core Consulting LLC Methods, systems, apparatuses and devices for facilitating motion analysis in an environment
KR102318555B1 (ko) 2020-03-19 2021-10-29 한국과학기술연구원 광소자용 역나노콘과 그 제조방법

Also Published As

Publication number Publication date
WO2010144740A1 (en) 2010-12-16
US20100324120A1 (en) 2010-12-23
MX342785B (es) 2016-10-12
MX2011013320A (es) 2012-02-28
DK3431076T3 (da) 2021-12-20
SG10201912450XA (en) 2020-03-30
KR20190065474A (ko) 2019-06-11
JP2017122126A (ja) 2017-07-13
KR102205886B1 (ko) 2021-01-21
HUE038796T2 (hu) 2018-11-28
MX367665B (es) 2019-08-30
AU2010259984A1 (en) 2012-01-12
PT2440183T (pt) 2018-10-30
ES2689168T3 (es) 2018-11-08
IL244945B (en) 2020-06-30
JP2012530059A (ja) 2012-11-29
IL290077A (en) 2022-03-01
KR20220038506A (ko) 2022-03-28
NZ712719A (en) 2017-03-31
KR102374518B1 (ko) 2022-03-16
US8802644B2 (en) 2014-08-12
SI2440183T1 (sl) 2018-10-30
KR102066189B1 (ko) 2020-01-14
KR20210008938A (ko) 2021-01-25
SG176786A1 (en) 2012-01-30
PL2440183T3 (pl) 2019-01-31
IL274826A (en) 2020-07-30
CN102625696B (zh) 2015-06-03
EP2440183A1 (en) 2012-04-18
US20120183602A1 (en) 2012-07-19
US9394234B2 (en) 2016-07-19
JP2015232048A (ja) 2015-12-24
EA201791744A2 (ru) 2018-03-30
EA024960B1 (ru) 2016-11-30
EA028860B1 (ru) 2018-01-31
IL244945A0 (en) 2016-05-31
KR20120081065A (ko) 2012-07-18
CA2764609A1 (en) 2010-12-16
JP6592144B2 (ja) 2019-10-16
NZ622843A (en) 2015-10-30
CY1124769T1 (el) 2022-11-25
US8158601B2 (en) 2012-04-17
HRP20211619T1 (hr) 2022-02-04
CY1120641T1 (el) 2019-12-11
CN102625696A (zh) 2012-08-01
HRP20181221T1 (hr) 2018-10-05
DK2440183T3 (en) 2018-10-01
KR20170091798A (ko) 2017-08-09
PT3431076T (pt) 2021-10-26
EP3431076B1 (en) 2021-10-06
US20150166465A1 (en) 2015-06-18
AU2017202702B2 (en) 2019-05-02
KR20200006176A (ko) 2020-01-17
AU2019204984B2 (en) 2021-01-28
SG10201403054SA (en) 2014-10-30
KR101987962B1 (ko) 2019-06-11
AU2017202702A1 (en) 2017-05-18
EP2440183B1 (en) 2018-07-18
JP5819291B2 (ja) 2015-11-24
JP2018141019A (ja) 2018-09-13
ES2901627T3 (es) 2022-03-23
AU2021201228A1 (en) 2021-03-11
LT3431076T (lt) 2021-10-25
LT2440183T (lt) 2018-08-10
HUE056773T2 (hu) 2022-03-28
EA201791744A3 (ru) 2018-07-31
TR201811076T4 (tr) 2018-08-27
AU2019204984A1 (en) 2019-08-01
EA201190306A1 (ru) 2013-01-30
CN104873464A (zh) 2015-09-02
IL216876A (en) 2016-04-21
CN104873464B (zh) 2018-06-22
JP6132321B2 (ja) 2017-05-24
SI3431076T1 (sl) 2022-04-29
EP2440183A4 (en) 2013-02-27
NZ596958A (en) 2014-04-30
KR20230098713A (ko) 2023-07-04
AU2010259984B2 (en) 2017-03-09
IL216876A0 (en) 2012-02-29
CA2764609C (en) 2018-10-02
MX2019010340A (es) 2019-10-14
AU2021201228B2 (en) 2022-07-07
EP3431076A1 (en) 2019-01-23
JP6359719B2 (ja) 2018-07-18
KR101766408B1 (ko) 2017-08-10
CA3014827A1 (en) 2010-12-16
US20170143631A1 (en) 2017-05-25
HK1212620A1 (zh) 2016-06-17
PL3431076T3 (pl) 2022-01-31

Similar Documents

Publication Publication Date Title
EA201791744A2 (ru) Улучшенная липидная композиция
CY1124924T1 (el) Φαρμακευτικη συνθεση καρβετοκινης
CY1119684T1 (el) Αντι-ιικες ενωσεις
EA201190242A1 (ru) Липидные композиции
EA201490596A1 (ru) Новые производные дигидрохинолин-2-она
EA201001680A1 (ru) Соединения бензолсульфонамидтиазола и оксазола
EA201490567A1 (ru) Новые бициклические производные дигидрохинолин-2-она
EA201500653A1 (ru) Композиции, содержащие триазольное соединение
EA201590887A1 (ru) Композиция
EA202190619A1 (ru) Противовирусные соединения
EA201071339A1 (ru) Фосфорсодержащие производные в качестве ингибиторов киназы
EA201270257A1 (ru) Производные n1-сульфонил-5-фторпиримидинона
EA201290183A1 (ru) Бензодиазепиновый ингибитор бромодомена
EA201170396A1 (ru) 2-оксо-1,2-дигидрохинолиновые модуляторы иммунной функции
EA200971140A1 (ru) Триарильные соединения и композиции, их содержащие
EA201490846A1 (ru) Новые производные арилхинолина
EA201171246A1 (ru) Содержащая трегалулозу композиция, ее получение и применение
EA201290642A1 (ru) Соединения и способы
EA201300443A1 (ru) Способ получения дигидроптеридинонов и их промежуточных продуктов
EA201101478A1 (ru) Композиции и способы модулирования связывания ретинола с ретинолсвязывающим белком 4 (rbp4)
EA201270253A1 (ru) Производные n1-ацил-5-фторпиримидинона
EA201270256A1 (ru) Производные т1-замещенного 5-фтор-2-оксопиримидинон-1 (2h) -карбоксамида
BR112014004523A2 (pt) composto e composição aromatizante
EA201400058A1 (ru) Соединения и композиции на основе амидопиридиловых эфиров и их применение против паразитов
EA201071367A1 (ru) Соли соединений ингибиторов вич