PT2419144T - Composição para a administração de material genético - Google Patents
Composição para a administração de material genéticoInfo
- Publication number
- PT2419144T PT2419144T PT10714058T PT10714058T PT2419144T PT 2419144 T PT2419144 T PT 2419144T PT 10714058 T PT10714058 T PT 10714058T PT 10714058 T PT10714058 T PT 10714058T PT 2419144 T PT2419144 T PT 2419144T
- Authority
- PT
- Portugal
- Prior art keywords
- delivery
- composition
- genetic material
- genetic
- Prior art date
Links
Classifications
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- 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
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- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
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- 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
- A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/04—Linear peptides containing only normal peptide links
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
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- 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.
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- 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
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- 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
-
- 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
- C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
- C12N2760/00011—Details
- C12N2760/20011—Rhabdoviridae
- C12N2760/20022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
- C12N2760/00011—Details
- C12N2760/20011—Rhabdoviridae
- C12N2760/20031—Uses of virus other than therapeutic or vaccine, e.g. disinfectant
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Inorganic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GB0906693A GB0906693D0 (en) | 2009-04-17 | 2009-04-17 | Composition and method |
GB0906692A GB0906692D0 (en) | 2009-04-17 | 2009-04-17 | Composition |
Publications (1)
Publication Number | Publication Date |
---|---|
PT2419144T true PT2419144T (pt) | 2019-09-16 |
Family
ID=42333482
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT10714058T PT2419144T (pt) | 2009-04-17 | 2010-04-15 | Composição para a administração de material genético |
Country Status (8)
Country | Link |
---|---|
US (6) | US20130053426A1 (pt) |
EP (3) | EP3569254B1 (pt) |
JP (1) | JP5713325B2 (pt) |
CN (1) | CN102497887A (pt) |
DK (1) | DK2419144T3 (pt) |
ES (2) | ES2927225T3 (pt) |
PT (1) | PT2419144T (pt) |
WO (1) | WO2010119256A1 (pt) |
Families Citing this family (82)
Publication number | Priority date | Publication date | Assignee | Title |
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US9085778B2 (en) | 2006-05-03 | 2015-07-21 | VL27, Inc. | Exosome transfer of nucleic acids to cells |
EP3569254B1 (en) | 2009-04-17 | 2022-07-20 | Oxford University Innovation Limited | Composition for delivery of genetic material |
ES2713852T3 (es) | 2009-12-01 | 2019-05-24 | Translate Bio Inc | Derivado de esteroides para la administración de ARNm en enfermedades genéticas humanas |
WO2011106376A2 (en) * | 2010-02-23 | 2011-09-01 | The General Hospital Corporation | Use of microvesicles in the treatment of medical conditions |
US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
WO2012091136A1 (ja) * | 2010-12-28 | 2012-07-05 | 学校法人東京医科大学 | 発現ベクター、それを用いたキャリアの製造方法およびその用途 |
WO2012162563A2 (en) | 2011-05-24 | 2012-11-29 | The Regents Of The University Of California | Method for exosomal biomarker detection by electric field-induced release and measurement |
IL280771B2 (en) | 2011-06-08 | 2024-03-01 | Shire Human Genetic Therapies | Preparations of lipid nanoparticles and methods for administration of mRNA |
GB201121070D0 (en) * | 2011-12-07 | 2012-01-18 | Isis Innovation | composition for delivery of biotherapeutics |
GB201121069D0 (en) * | 2011-12-07 | 2012-01-18 | Isis Innovation | Delivery system |
EP3336190B1 (en) * | 2011-12-13 | 2021-02-24 | Henry Ford Health System | Methods, systems, and compositions for cell-derived/vesicle-based microrna delivery |
US10227593B2 (en) | 2011-12-13 | 2019-03-12 | Henry Ford Health System | Methods, systems, and compositions for cell-derived/vesicle-based microRNA delivery |
ES2691296T3 (es) * | 2012-04-03 | 2018-11-26 | Reneuron Limited | Micropartículas de células madre |
EP2859102A4 (en) | 2012-06-08 | 2016-05-11 | Shire Human Genetic Therapies | NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF |
JP6272765B2 (ja) * | 2012-10-01 | 2018-01-31 | 国立大学法人京都大学 | ナノゲル/エキソソーム複合体とdds |
CN103865942A (zh) * | 2012-12-18 | 2014-06-18 | 常州碳宇纳米科技有限公司 | 一种可提高基因转染效率的纳米粒子和基于该粒子的基因转染试剂的制备 |
US10308959B2 (en) | 2013-01-18 | 2019-06-04 | Henry Ford Health System | Methods, systems, and compositions relating to MiRNA-146a |
WO2014159662A1 (en) | 2013-03-13 | 2014-10-02 | University Of Miami | Method for isolation and purification of microvesicles from cell culture supernatants and biological fluids |
KR20210122917A (ko) | 2013-03-14 | 2021-10-12 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
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JP6137894B2 (ja) * | 2013-03-22 | 2017-05-31 | 国立大学法人京都大学 | リポソーム−エキソソームハイブリッドベシクル及びその調製法 |
JP6542197B2 (ja) * | 2013-04-12 | 2019-07-10 | エヴォックス・セラピューティクス・リミテッド | 治療的送達小胞 |
US10538570B2 (en) * | 2013-09-30 | 2020-01-21 | Northwestern University | Targeted and modular exosome loading system |
EA034103B1 (ru) | 2013-10-22 | 2019-12-27 | Транслейт Био, Инк. | СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК |
US11224642B2 (en) | 2013-10-22 | 2022-01-18 | Translate Bio, Inc. | MRNA therapy for argininosuccinate synthetase deficiency |
CN104726410B (zh) * | 2013-12-23 | 2019-09-17 | 浙江大学 | 一种具有免疫抑制功能的外排体及其应用 |
KR102541164B1 (ko) | 2014-01-21 | 2023-06-08 | 안자리움 바이오사이언시스 아게 | 하이브리도좀, 이를 포함하는 조성물, 이의 제조 방법 및 이의 용도 |
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WO2016179417A2 (en) * | 2015-05-06 | 2016-11-10 | The University Of Utah Research Foundation | Exosome delivery of micrornas |
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CN112972702A (zh) * | 2019-12-17 | 2021-06-18 | 南京大学 | 用于治疗耐药菌感染的外泌体制剂 |
CA3164176A1 (en) | 2020-01-13 | 2021-07-22 | Carmine Therapeutics Pte. Ltd. | Nucleic acid loaded red blood cell extracellular vesicles |
EP4097474A4 (en) | 2020-01-27 | 2024-03-27 | Mantra Bio Inc | NON-NATURALLY OCCURRING VESICLES COMPRISING A CHIMERIC VESICLE LOCALIZATION COMPONENT, METHODS OF PREPARATION AND USES THEREOF |
EP4132479A1 (en) | 2020-04-07 | 2023-02-15 | Ramot at Tel-Aviv University Ltd. | Cannabidiol-containing compositions and uses thereof |
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US20160067355A1 (en) | 2016-03-10 |
JP2012524052A (ja) | 2012-10-11 |
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WO2010119256A1 (en) | 2010-10-21 |
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US20170182182A1 (en) | 2017-06-29 |
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