IL292379A - Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders - Google Patents
Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disordersInfo
- Publication number
- IL292379A IL292379A IL292379A IL29237922A IL292379A IL 292379 A IL292379 A IL 292379A IL 292379 A IL292379 A IL 292379A IL 29237922 A IL29237922 A IL 29237922A IL 292379 A IL292379 A IL 292379A
- Authority
- IL
- Israel
- Prior art keywords
- nucleic acid
- vector
- cfh
- seq
- aav
- Prior art date
Links
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Classifications
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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
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- 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
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- 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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- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
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- Biophysics (AREA)
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- Ophthalmology & Optometry (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
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- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962924338P | 2019-10-22 | 2019-10-22 | |
| PCT/US2020/056862 WO2021081203A1 (en) | 2019-10-22 | 2020-10-22 | Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL292379A true IL292379A (en) | 2022-06-01 |
Family
ID=75586875
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL292379A IL292379A (en) | 2019-10-22 | 2020-10-22 | Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20210123076A1 (https=) |
| EP (1) | EP4048321A4 (https=) |
| JP (1) | JP2022553309A (https=) |
| KR (1) | KR20230019062A (https=) |
| CN (1) | CN115175706A (https=) |
| AU (1) | AU2020371663A1 (https=) |
| CA (1) | CA3158517A1 (https=) |
| IL (1) | IL292379A (https=) |
| MX (1) | MX2022004770A (https=) |
| WO (1) | WO2021081203A1 (https=) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| US7745389B2 (en) * | 2005-02-14 | 2010-06-29 | University Of Iowa Research Foundation | Methods for treatment of age-related macular degeneration |
| CN101160412A (zh) * | 2005-02-14 | 2008-04-09 | 爱荷华大学研究基金会 | 治疗和诊断年龄相关性黄斑变性的方法和试剂 |
| WO2007127428A2 (en) * | 2006-04-28 | 2007-11-08 | University Of Florida Research Foundation, Inc. | Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery |
| EP2479284B1 (en) * | 2006-07-13 | 2017-09-20 | University of Iowa Research Foundation | Methods and reagents for treatment and diagnosis of vascular disorders and age-related macular degeneration |
| CN108291216B (zh) * | 2015-09-24 | 2022-09-16 | 宾夕法尼亚州大学信托人 | 用于治疗补体介导的疾病的组合物和方法 |
| WO2017114401A1 (zh) * | 2015-12-31 | 2017-07-06 | 江苏匡亚生物医药科技有限公司 | 具有补体调节活性的重组补体因子h-免疫球蛋白融合蛋白及其制备方法与应用 |
| GB201706808D0 (en) * | 2017-04-28 | 2017-06-14 | Univ Newcastle | Modified complement proteins and uses thereof |
| EP3697920A4 (en) * | 2017-10-20 | 2022-03-02 | Gemini Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION |
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2020
- 2020-10-22 EP EP20879947.8A patent/EP4048321A4/en active Pending
- 2020-10-22 AU AU2020371663A patent/AU2020371663A1/en not_active Abandoned
- 2020-10-22 CA CA3158517A patent/CA3158517A1/en active Pending
- 2020-10-22 KR KR1020227017063A patent/KR20230019062A/ko not_active Ceased
- 2020-10-22 WO PCT/US2020/056862 patent/WO2021081203A1/en not_active Ceased
- 2020-10-22 CN CN202080089391.2A patent/CN115175706A/zh active Pending
- 2020-10-22 MX MX2022004770A patent/MX2022004770A/es unknown
- 2020-10-22 IL IL292379A patent/IL292379A/en unknown
- 2020-10-22 US US17/077,683 patent/US20210123076A1/en active Pending
- 2020-10-22 JP JP2022523415A patent/JP2022553309A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4048321A4 (en) | 2023-11-22 |
| WO2021081203A1 (en) | 2021-04-29 |
| US20210123076A1 (en) | 2021-04-29 |
| CA3158517A1 (en) | 2021-04-29 |
| AU2020371663A1 (en) | 2022-05-19 |
| MX2022004770A (es) | 2022-10-07 |
| EP4048321A1 (en) | 2022-08-31 |
| KR20230019062A (ko) | 2023-02-07 |
| JP2022553309A (ja) | 2022-12-22 |
| CN115175706A (zh) | 2022-10-11 |
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