JP7731909B2 - Ａａｖカプシド変異型及びそれを含むａａｖ粒子 - Google Patents
Ａａｖカプシド変異型及びそれを含むａａｖ粒子Info
- Publication number
- JP7731909B2 JP7731909B2 JP2022568852A JP2022568852A JP7731909B2 JP 7731909 B2 JP7731909 B2 JP 7731909B2 JP 2022568852 A JP2022568852 A JP 2022568852A JP 2022568852 A JP2022568852 A JP 2022568852A JP 7731909 B2 JP7731909 B2 JP 7731909B2
- Authority
- JP
- Japan
- Prior art keywords
- seq
- aav capsid
- amino acid
- acid sequence
- aav
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
-
- 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
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C40—COMBINATORIAL TECHNOLOGY
- C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
- C40B40/00—Libraries per se, e.g. arrays, mixtures
- C40B40/04—Libraries containing only organic compounds
- C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
- C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
-
- 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
- C12N2750/14122—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
- 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
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025136742A JP2025173511A (ja) | 2020-05-13 | 2025-08-20 | Ａａｖカプシドの指向性の再指示 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063023927P | 2020-05-13 | 2020-05-13 | |
| US63/023,927 | 2020-05-13 | ||
| US202063122300P | 2020-12-07 | 2020-12-07 | |
| US63/122,300 | 2020-12-07 | ||
| PCT/US2021/025061 WO2021230987A1 (en) | 2020-05-13 | 2021-03-31 | Redirection of tropism of aav capsids |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025136742A Division JP2025173511A (ja) | 2020-05-13 | 2025-08-20 | Ａａｖカプシドの指向性の再指示 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023525810A JP2023525810A (ja) | 2023-06-19 |
| JP2023525810A5 JP2023525810A5 (enExample) | 2024-04-09 |
| JP7731909B2 true JP7731909B2 (ja) | 2025-09-01 |
Family
ID=75639982
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022568852A Active JP7731909B2 (ja) | 2020-05-13 | 2021-03-31 | Ａａｖカプシド変異型及びそれを含むａａｖ粒子 |
| JP2025136742A Pending JP2025173511A (ja) | 2020-05-13 | 2025-08-20 | Ａａｖカプシドの指向性の再指示 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025136742A Pending JP2025173511A (ja) | 2020-05-13 | 2025-08-20 | Ａａｖカプシドの指向性の再指示 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US20230203102A1 (enExample) |
| EP (1) | EP4149955A1 (enExample) |
| JP (2) | JP7731909B2 (enExample) |
| KR (1) | KR20230022175A (enExample) |
| CN (1) | CN116096734A (enExample) |
| AU (1) | AU2021273447B2 (enExample) |
| BR (1) | BR112022023106A2 (enExample) |
| CA (1) | CA3182970A1 (enExample) |
| CO (1) | CO2022016156A2 (enExample) |
| IL (1) | IL298001A (enExample) |
| MX (1) | MX2022014256A (enExample) |
| PE (1) | PE20230767A1 (enExample) |
| TW (1) | TWI900553B (enExample) |
| WO (1) | WO2021230987A1 (enExample) |
Families Citing this family (80)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2525067T3 (es) * | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| JP2022513067A (ja) * | 2018-11-16 | 2022-02-07 | アスクレピオス バイオファーマシューティカル， インコーポレイテッド | ポンペ病を処置するための治療用アデノ随伴ウイルス |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| US20230295238A1 (en) * | 2020-07-23 | 2023-09-21 | The Scripps Research Institute | Insulin receptor-mediated enhancement of gene transfer |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| JP2024533174A (ja) * | 2021-09-03 | 2024-09-12 | ビオマリン プハルマセウトイカル インコーポレイテッド | Ａａｖカプシド組成物及び送達方法 |
| WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20250049955A1 (en) | 2021-11-17 | 2025-02-13 | Voyager Therapeutics, Inc. | Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| WO2023092002A2 (en) * | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord |
| EP4433169A1 (en) | 2021-11-17 | 2024-09-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20250034559A1 (en) | 2021-11-17 | 2025-01-30 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of tau-related disorders |
| WO2023147374A2 (en) | 2022-01-25 | 2023-08-03 | Voyager Therapeutics, Inc. | Baculovirus expression system |
| US20250297280A2 (en) * | 2022-02-01 | 2025-09-25 | The Broad Institute, Inc. | Adeno-associated viral vectors and uses thereof |
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| JP2025528068A (ja) | 2022-08-03 | 2025-08-26 | ボイジャー セラピューティクス インコーポレイテッド | 血液脳関門を通過させるための組成物及び方法 |
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| WO2024055020A2 (en) * | 2022-09-09 | 2024-03-14 | The Regents Of The University Of California | Reprogramming tropism via displayed peptides tiling receptor-ligands |
| KR20250069606A (ko) | 2022-09-15 | 2025-05-19 | 보이저 테라퓨틱스, 인크. | 타우 결합 화합물 |
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| US20230203102A1 (en) | 2023-06-29 |
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