MX2021003015A - Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis. - Google Patents

Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.

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Publication number
MX2021003015A
MX2021003015A MX2021003015A MX2021003015A MX2021003015A MX 2021003015 A MX2021003015 A MX 2021003015A MX 2021003015 A MX2021003015 A MX 2021003015A MX 2021003015 A MX2021003015 A MX 2021003015A MX 2021003015 A MX2021003015 A MX 2021003015A
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MX
Mexico
Prior art keywords
phosphatase
glucose
treatment
storage disease
glycogen storage
Prior art date
Application number
MX2021003015A
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English (en)
Inventor
Vladimir Presnyak
Paolo G V Martini
Athanasios Dousis
Jingsong Cao
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Modernatx Inc
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Filing date
Publication date
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of MX2021003015A publication Critical patent/MX2021003015A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Medicinal Chemistry (AREA)
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  • Dermatology (AREA)
  • Nanotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Optics & Photonics (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente descripción se refiere a terapia con ARNm para el tratamiento de la glucogenosis tipo 1a, (GSD-Ia) y síntomas relacionados tales como la hipoglucemia. Los ARNm para uso en la invención, cuando se administran in vivo, codifican glucosa-6-fosfatasa humana (G6Pase o G6PC), y fragmentos o variantes funcionales de esta. Los ARNm de la invención preferiblemente se encapsulan en nanopartículas lipídicas (LNP) para lograr el suministro eficaz a células y/o tejidos en sujetos, cuando se administran a estos. Las terapias con ARNm de la invención aumentan y/o restauran los niveles deficientes de la expresión y/o actividad de G6PC en sujetos. Las terapias con ARNm de la invención, además, aumentan la producción de glucosa y reducen la acumulación anormal de glucógeno y/o glucosa-6-fosfato asociada a GSDIa.
MX2021003015A 2018-09-13 2019-09-12 Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis. MX2021003015A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862730940P 2018-09-13 2018-09-13
US201962812800P 2019-03-01 2019-03-01
PCT/US2019/050827 WO2020056147A2 (en) 2018-09-13 2019-09-12 Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease

Publications (1)

Publication Number Publication Date
MX2021003015A true MX2021003015A (es) 2021-07-15

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021003015A MX2021003015A (es) 2018-09-13 2019-09-12 Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.

Country Status (9)

Country Link
US (1) US20230009009A1 (es)
EP (1) EP3849595A2 (es)
JP (1) JP2022500436A (es)
CN (1) CN113164561A (es)
AU (1) AU2019337637A1 (es)
CA (1) CA3112208A1 (es)
MA (1) MA53609A (es)
MX (1) MX2021003015A (es)
WO (1) WO2020056147A2 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021247535A1 (en) * 2020-06-01 2021-12-09 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
US20230242908A1 (en) * 2020-06-23 2023-08-03 Modernatx, Inc. Lnp compositions comprising mrna therapeutics with extended half-life
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase 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)
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
CN116589435A (zh) * 2022-06-20 2023-08-15 成都威斯津生物医药科技有限公司 用于核酸递送的可电离脂质及其组合物
CN117771354A (zh) * 2024-02-23 2024-03-29 云南省畜牧兽医科学院 一种预防O型***的mRNA-LNP疫苗及其制备方法

Family Cites Families (127)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
US5563250A (en) 1987-12-02 1996-10-08 Neorx Corporation Cleavable conjugates for the delivery and release of agents in native form
US5505931A (en) 1993-03-04 1996-04-09 The Dow Chemical Company Acid cleavable compounds, their preparation and use as bifunctional acid-labile crosslinking agents
AU692196B2 (en) 1993-11-26 1998-06-04 British Technology Group Limited Translational enhancer DNA
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6004573A (en) 1997-10-03 1999-12-21 Macromed, Inc. Biodegradable low molecular weight triblock poly(lactide-co-glycolide) polyethylene glycol copolymers having reverse thermal gelation properties
US6517869B1 (en) 1997-12-12 2003-02-11 Expression Genetics, Inc. Positively charged poly(alpha-(omega-aminoalkyl)lycolic acid) for the delivery of a bioactive agent via tissue and cellular uptake
AU1819499A (en) 1997-12-12 1999-06-28 Samyang Corporation Positively-charged poly{alpha-(omega-aminoalkyl)glycolic acid} for the delivery of a bioactive agent via tissue and cellular uptake
WO1999034831A1 (en) 1998-01-05 1999-07-15 University Of Washington Enhanced transport using membrane disruptive agents
US6426086B1 (en) 1998-02-03 2002-07-30 The Regents Of The University Of California pH-sensitive, serum-stable liposomes
CN1333678A (zh) 1998-05-20 2002-01-30 表达遗传学公司 定向肝细胞的聚乙二醇接枝的聚－l－赖氨酸的聚合基因载体
US7091192B1 (en) 1998-07-01 2006-08-15 California Institute Of Technology Linear cyclodextrin copolymers
KR20010079526A (ko) 1998-07-13 2001-08-22 추후제출 용해성 생분해 유전자 전달 담체인 폴리-ｌ-리신의폴리에스테르 유사체
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
WO2000075164A1 (en) 1999-06-07 2000-12-14 Mirus Corporation COMPOSITIONS AND METHODS FOR DRUG DELIVERY USING pH SENSITIVE MOLECULES
AU2764801A (en) 2000-01-07 2001-07-24 University Of Washington Enhanced transport of agents using membrane disruptive agents
US6696038B1 (en) 2000-09-14 2004-02-24 Expression Genetics, Inc. Cationic lipopolymer as biocompatible gene delivery agent
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US6897196B1 (en) 2001-02-07 2005-05-24 The Regents Of The University Of California pH sensitive lipids based on ortho ester linkers, composition and method
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US6586524B2 (en) 2001-07-19 2003-07-01 Expression Genetics, Inc. Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier
EP1412065A2 (en) 2001-07-27 2004-04-28 President And Fellows Of Harvard College Laminar mixing apparatus and methods
EP2428569B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
WO2003092665A2 (en) 2002-05-02 2003-11-13 Massachusetts Eye And Ear Infirmary Ocular drug delivery systems and use thereof
WO2005007819A2 (en) 2003-07-09 2005-01-27 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
WO2005062854A2 (en) 2003-12-19 2005-07-14 University Of Cincinnati Polyamides for nucleic acid delivery
NZ550320A (en) 2004-04-15 2010-02-26 Chiasma Inc Compositions capable of facilitating penetration across a biological barrier
US20050261233A1 (en) * 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
US8057821B2 (en) 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
US8354476B2 (en) 2004-12-10 2013-01-15 Kala Pharmaceuticals, Inc. Functionalized poly(ether-anhydride) block copolymers
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006110776A2 (en) 2005-04-12 2006-10-19 Nektar Therapeutics Al, Corporation Polyethylene glycol cojugates of antimicrobial agents
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
RU2008103346A (ru) 2005-06-30 2009-08-10 Аркемикс Корп. (Us) Материалы и способы для получения полностью 2-модифицированных транскриптов нуклеиновых кислот
PL2578685T3 (pl) * 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
WO2007026249A2 (en) 2005-09-01 2007-03-08 Novartis Vaccines And Diagnostics Gmbh & Co Kg Multiple vaccination including serogroup c meningococcus
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP1968643A2 (en) 2005-12-16 2008-09-17 Diatos Cell penetrating peptide conjugates for delivering of nucleic acids into a cell
KR100859972B1 (ko) 2006-02-20 2008-09-25 이화여자대학교 산학협력단 막투과 단백질 도메인 펩타이드
US8309680B2 (en) 2006-02-21 2012-11-13 Nektar Therapeutics Segmented degradable polymers and conjugates made therefrom
CN101583379B (zh) 2006-10-05 2013-04-03 约翰斯霍普金斯大学 使用优良聚合物纳米粒子的水溶性差药物的水可分散性口服，肠胃外和局部制剂
CA2670749A1 (en) 2006-12-05 2008-06-12 Landec Corporation Drug delivery
CA2671925A1 (en) 2006-12-21 2008-07-10 Stryker Corporation Sustained-release formulations comprising crystals, macromolecular gels, and particulate suspensions of biologic agents
WO2008078180A2 (en) 2006-12-22 2008-07-03 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
KR101442867B1 (ko) 2006-12-27 2014-09-25 넥타르 테라퓨틱스 제거될 수 있는 결합을 지니는 인자 ⅸ 부분­중합체 컨주게이트
WO2008103276A2 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
AU2008234364B2 (en) 2007-03-30 2013-10-17 Altum-Avro Pharma Partnership Biphasic lipid-vesicle composition and method for treating cervical dysplasia by intravaginal delivery
CA2686735A1 (en) 2007-05-04 2008-11-13 Mdrna, Inc. Amino acid lipids and uses thereof
EP3156414B1 (en) 2007-09-26 2019-12-04 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
ES2647538T3 (es) 2007-09-28 2017-12-22 Pfizer Inc. Direccionamiento a células de cáncer usando nanopartículas
US20100004315A1 (en) 2008-03-14 2010-01-07 Gregory Slobodkin Biodegradable Cross-Linked Branched Poly(Alkylene Imines)
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
US8613951B2 (en) 2008-06-16 2013-12-24 Bind Therapeutics, Inc. Therapeutic polymeric nanoparticles with mTor inhibitors and methods of making and using same
HUE035770T2 (en) 2008-06-16 2018-05-28 Pfizer Process for the preparation of diblock copolymers functionalized with targeting material for use in the preparation of therapeutic nanoparticles
EP2309991B1 (en) 2008-06-16 2019-03-06 Pfizer Inc Therapeutic polymeric nanoparticles comprising vinca alkaloids and methods of making and using same
PT2774608T (pt) 2008-06-16 2020-01-17 Pfizer Nanopartículas poliméricas carregadas com fármaco e métodos de produção e utilização das mesmas
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
JP2012512175A (ja) 2008-12-15 2012-05-31 バインド バイオサイエンシズ インコーポレイテッド 治療薬を徐放するための長時間循環性ナノ粒子
WO2010087791A1 (en) 2009-01-27 2010-08-05 Utc Power Corporation Distributively cooled, integrated water-gas shift reactor and vaporizer
WO2010108108A2 (en) 2009-03-20 2010-09-23 Egen, Inc. Polyamine derivatives
CN102625696B (zh) 2009-06-10 2015-06-03 阿尔尼拉姆医药品有限公司 改进的脂质制剂
CA2816925C (en) 2009-11-04 2023-01-10 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
WO2011062965A2 (en) 2009-11-18 2011-05-26 University Of Washington Through Its Center For Commercialization Targeting monomers and polymers having targeting blocks
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
EP2512487A4 (en) 2009-12-15 2013-08-07 THERAPEUTIC POLYMERNANOPARTICLES WITH CORTICOSTEROIDS AND METHOD FOR THE PRODUCTION AND USE THEREOF
EA201290498A1 (ru) 2009-12-15 2013-01-30 Байнд Байосайенсиз, Инк. Терапевтические полимерные наночастицы, включающие эпотилон, и способы их получения и применения
US9295649B2 (en) 2009-12-15 2016-03-29 Bind Therapeutics, Inc. Therapeutic polymeric nanoparticle compositions with high glass transition temperature or high molecular weight copolymers
US20130231287A1 (en) 2010-02-25 2013-09-05 Parimala Nacharaju Pegylated albumin polymers and uses thereof
WO2011127255A1 (en) 2010-04-08 2011-10-13 Merck Sharp & Dohme Corp. Preparation of lipid nanoparticles
EP2555752B1 (en) 2010-04-09 2019-06-26 Pacira Pharmaceuticals, Inc. Method for formulating multivesicular liposomes
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
AU2011267078B2 (en) 2010-06-14 2014-09-25 F. Hoffmann-La Roche Ag Cell-penetrating peptides and uses therof
WO2011163483A2 (en) 2010-06-25 2011-12-29 Massachusetts Institute Of Technology Polymers for biomaterials and therapeutics
ES2649896T3 (es) 2010-07-06 2018-01-16 Glaxosmithkline Biologicals Sa Emulsiones catiónicas de aceite en agua
CN103153284B (zh) 2010-07-06 2015-11-25 诺华股份有限公司 含有其pKa值有利于RNA递送的脂质的脂质体
RS63817B1 (sr) 2010-07-06 2023-01-31 Glaxosmithkline Biologicals Sa Čestice za isporuku slične virionu za molekule samoreplicirajuće rnk
WO2012012772A2 (en) 2010-07-22 2012-01-26 The Johns Hopkins University Drug eluting hydrogels for catheter delivery
CA2801523C (en) 2010-07-30 2021-08-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
US9121065B2 (en) 2010-08-09 2015-09-01 The Trustees Of The University Of Pennsylvania Nanoparticle-oligonucleotide hybrid structures and methods of use thereof
US20130195799A1 (en) 2010-08-19 2013-08-01 Peg Biosciences, Inc. Synergistic biomolecule-polymer conjugates
AU2011291582A1 (en) 2010-08-20 2013-03-07 Cerulean Pharma Inc. Conjugates, particles, compositions, and related methods
BR112013004879A2 (pt) 2010-08-31 2018-04-24 Novartis Ag lipossomas pequenos para entrega de rna que codifica imunogénio
HUE058667T2 (hu) 2010-08-31 2022-09-28 Glaxosmithkline Biologicals Sa Pegilált liposzómák immunogént kódoló RNS szállítására
EP2611420B1 (en) 2010-08-31 2019-03-27 GlaxoSmithKline Biologicals SA Lipids suitable for liposomal delivery of protein-coding rna
US10307372B2 (en) 2010-09-10 2019-06-04 The Johns Hopkins University Rapid diffusion of large polymeric nanoparticles in the mammalian brain
CN103269722A (zh) 2010-09-24 2013-08-28 马林克罗特有限公司 治疗性和/或诊断性纳米载体的靶向的适体缀合物
EA201390600A1 (ru) 2010-10-22 2013-09-30 Байнд Терапьютикс, Инк. Терапевтические наночастицы с сополимерами с большим молекулярным весом
DK2663548T3 (en) 2011-01-11 2017-07-24 Alnylam Pharmaceuticals Inc PEGYLED LIPIDS AND THEIR USE FOR PHARMACEUTICAL SUPPLY
WO2012109121A1 (en) 2011-02-07 2012-08-16 Purdue Research Foundation Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2489371A1 (en) 2011-02-18 2012-08-22 Instituto Nacional de Investigacion y Tecnologia Agraria y Alimentaria Carrier peptides for drug delivery
US8846850B2 (en) 2011-02-22 2014-09-30 Rutgers, The State University Of New Jersey Amphiphilic macromolecules for nucleic acid delivery
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
ME03491B (me) 2011-06-08 2020-01-20 Translate Bio Inc Kompozicije lipidnih nanočestica i postupci za isporuku irnk
WO2012170889A1 (en) 2011-06-08 2012-12-13 Shire Human Genetic Therapies, Inc. Cleavable lipids
KR20140067070A (ko) 2011-08-31 2014-06-03 말린크로트 엘엘씨 Ｈ-포스포네이트에 의한 나노입자 ｐｅｇ 개질
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS AND METHODS OF USE
HUE057725T2 (hu) 2011-10-03 2022-06-28 Modernatx Inc Módosított nukleozidok, nukleotidok és nukleinsavak és ezek felhasználása
WO2013082111A2 (en) 2011-11-29 2013-06-06 The University Of North Carolina At Chapel Hill Geometrically engineered particles and methods for modulating macrophage or immune responses
HUE21212055T1 (hu) 2011-12-07 2022-11-28 Alnylam Pharmaceuticals Inc Biológiailag lebontható lipidek hatóanyagok bejuttatására
EP2788316B1 (en) 2011-12-07 2019-04-24 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
EP2787977A4 (en) 2011-12-09 2015-05-06 Univ California LIPOSOMAL ACTIVE INVERTING
WO2013106073A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013106072A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013106086A1 (en) 2012-01-10 2013-07-18 Sorbent Therapeutics, Inc. Compositions comprising crosslinked cation-binding polymers and uses thereof
WO2013105101A1 (en) 2012-01-13 2013-07-18 Department Of Biotechnology Solid lipid nanoparticles entrapping hydrophilic/ amphiphilic drug and a process for preparing the same
EP2804632B1 (en) 2012-01-19 2019-09-18 The Johns Hopkins University Nanoparticle formulations with enhanced mucosal penetration
WO2013116126A1 (en) 2012-02-01 2013-08-08 Merck Sharp & Dohme Corp. Novel low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
JP6356614B2 (ja) 2012-02-17 2018-07-11 ユニバーシティ・オブ・ジョージア・リサーチ・ファウンデイション・インコーポレイテッド 薬剤のミトコンドリア輸送のためのナノ粒子
CN104394853B (zh) 2012-02-19 2017-10-10 纳维基因股份有限公司 多孔性纳米结构在递送中的用途
WO2013124867A1 (en) 2012-02-21 2013-08-29 Amrita Vishwa Vidyapeetham University Polymer - polymer or polymer - protein core - shell nano medicine loaded with multiple drug molecules
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
AU2013243947A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
US20150307542A1 (en) 2012-10-03 2015-10-29 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
WO2015164773A1 (en) * 2014-04-25 2015-10-29 Shire Human Genetic Therapies, Inc. Methods for purification of messenger rna
JP2017019215A (ja) * 2015-07-13 2017-01-26 富士フイルム株式会社 着色積層体およびその製造方法
WO2017100551A1 (en) * 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
WO2019023179A1 (en) * 2017-07-24 2019-01-31 Modernatx, Inc. MODIFIED mRNA ENCODING GLUCOSE-6-PHOSPHATASE AND USES THEREOF

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