JP2022553309A - 加齢黄斑変性ならびに他の眼疾患および障害の治療のためのアデノ随伴ウイルス(aav)ベクター - Google Patents
加齢黄斑変性ならびに他の眼疾患および障害の治療のためのアデノ随伴ウイルス(aav)ベクター Download PDFInfo
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- JP2022553309A JP2022553309A JP2022523415A JP2022523415A JP2022553309A JP 2022553309 A JP2022553309 A JP 2022553309A JP 2022523415 A JP2022523415 A JP 2022523415A JP 2022523415 A JP2022523415 A JP 2022523415A JP 2022553309 A JP2022553309 A JP 2022553309A
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- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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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
- C12N15/63—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
-
- 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
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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
- A61K48/0075—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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- 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
-
- 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
-
- 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
-
- 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)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Ophthalmology & Optometry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962924338P | 2019-10-22 | 2019-10-22 | |
| US62/924,338 | 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2022553309A true JP2022553309A (ja) | 2022-12-22 |
| JP2022553309A5 JP2022553309A5 (https=) | 2023-10-30 |
Family
ID=75586875
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523415A Pending JP2022553309A (ja) | 2019-10-22 | 2020-10-22 | 加齢黄斑変性ならびに他の眼疾患および障害の治療のためのアデノ随伴ウイルス(aav)ベクター |
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=) |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2018527941A (ja) * | 2015-09-24 | 2018-09-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 補体媒介性疾患を処置するための組成物及び方法 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
-
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
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2018527941A (ja) * | 2015-09-24 | 2018-09-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 補体媒介性疾患を処置するための組成物及び方法 |
Non-Patent Citations (2)
| Title |
|---|
| CLINICAL AND EXPERIMENTAL IMMUNOLOGY,, vol. 151, JPN6024040334, 2008, pages 14 - 24, ISSN: 0005432706 * |
| FRONTIERS IN GENETICS, vol. Volume 10, Article 465, JPN6024040335, May 2019 (2019-05-01), pages 1 - 6, ISSN: 0005613931 * |
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 |
| IL292379A (en) | 2022-06-01 |
| KR20230019062A (ko) | 2023-02-07 |
| CN115175706A (zh) | 2022-10-11 |
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