JP7834725B2 - ペプチド改変されたａａｖカプシド - Google Patents
ペプチド改変されたａａｖカプシドInfo
- Publication number
- JP7834725B2 JP7834725B2 JP2023515813A JP2023515813A JP7834725B2 JP 7834725 B2 JP7834725 B2 JP 7834725B2 JP 2023515813 A JP2023515813 A JP 2023515813A JP 2023515813 A JP2023515813 A JP 2023515813A JP 7834725 B2 JP7834725 B2 JP 7834725B2
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- muscle
- aav
- capsid protein
- aav9
- 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
-
- 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
- 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
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20306005 | 2020-09-10 | ||
| EP20306005.8 | 2020-09-10 | ||
| PCT/EP2021/074964 WO2022053630A1 (en) | 2020-09-10 | 2021-09-10 | Peptide-modified aav capsid |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2023544683A JP2023544683A (ja) | 2023-10-25 |
| JP2023544683A5 JP2023544683A5 (https=) | 2024-09-19 |
| JPWO2022053630A5 JPWO2022053630A5 (https=) | 2024-09-19 |
| JP7834725B2 true JP7834725B2 (ja) | 2026-03-24 |
Family
ID=72717827
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023515813A Active JP7834725B2 (ja) | 2020-09-10 | 2021-09-10 | ペプチド改変されたａａｖカプシド |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240209030A1 (https=) |
| EP (2) | EP4211231B1 (https=) |
| JP (1) | JP7834725B2 (https=) |
| CN (1) | CN116096431A (https=) |
| CA (1) | CA3193128A1 (https=) |
| DK (1) | DK4211231T3 (https=) |
| ES (1) | ES2984259T3 (https=) |
| FI (1) | FI4211231T3 (https=) |
| PL (1) | PL4211231T3 (https=) |
| PT (1) | PT4211231T (https=) |
| WO (1) | WO2022053630A1 (https=) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL297391A (en) | 2020-04-20 | 2022-12-01 | Tenaya Therapeutics Inc | An adeno-associated virus with an engineered capsid |
| EP4314295A1 (en) | 2021-03-26 | 2024-02-07 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| CN117897492A (zh) | 2021-08-04 | 2024-04-16 | 吉尼松公司 | 用于肌肉和cns中的基因表达的杂合启动子 |
| US20250249052A1 (en) * | 2022-04-07 | 2025-08-07 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
| CN116925192B (zh) * | 2022-04-12 | 2025-04-29 | 合肥星眸生物科技有限公司 | 一种融合型腺相关病毒及其应用 |
| JP2025524363A (ja) | 2022-06-09 | 2025-07-30 | ジェネトン | 糖原病ｉｉｉの処置のためのｎ末端短縮型ｇｄｅ |
| EP4707399A2 (en) | 2022-06-10 | 2026-03-11 | Généthon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
| US20260053923A1 (en) | 2022-10-07 | 2026-02-26 | Genethon | Immunotherapy of Skeletal Myopathies Using ANTI-FAP CAR-T Cells |
| EP4609882A1 (en) * | 2022-10-12 | 2025-09-03 | CPC Corporation | Gene introduction method, gene therapy method, and tissue regeneration method |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| EP4677073A1 (en) | 2023-03-10 | 2026-01-14 | Genethon | Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter |
| CN121420060A (zh) | 2023-06-29 | 2026-01-27 | 吉尼松公司 | 用于治疗糖原贮积病iii的n-端截短的糖原脱支酶 |
| WO2025048709A1 (en) * | 2023-08-25 | 2025-03-06 | Agency For Science, Technology And Research | Composition and methods for identification of novel aav variants for differentiated gene delivery to cardiomyoctes and/or skeletal myofibers |
| WO2025133247A1 (en) | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| KR102933125B1 (ko) * | 2024-02-01 | 2026-03-04 | 주식회사 글루진테라퓨틱스 | 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터 |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2026017727A1 (en) | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
| WO2026064428A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064444A1 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015006743A1 (en) | 2013-07-12 | 2015-01-15 | The Children's Hospital Of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| WO2019193119A1 (en) | 2018-04-05 | 2019-10-10 | Genethon | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
| WO2019207132A1 (en) | 2018-04-27 | 2019-10-31 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PE20190401A1 (es) * | 2016-07-26 | 2019-03-13 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado |
-
2021
- 2021-09-10 PL PL21777271.4T patent/PL4211231T3/pl unknown
- 2021-09-10 EP EP21777271.4A patent/EP4211231B1/en active Active
- 2021-09-10 DK DK21777271.4T patent/DK4211231T3/da active
- 2021-09-10 CN CN202180055064.XA patent/CN116096431A/zh active Pending
- 2021-09-10 JP JP2023515813A patent/JP7834725B2/ja active Active
- 2021-09-10 ES ES21777271T patent/ES2984259T3/es active Active
- 2021-09-10 US US18/024,103 patent/US20240209030A1/en active Pending
- 2021-09-10 EP EP24183857.2A patent/EP4421179A3/en not_active Withdrawn
- 2021-09-10 PT PT217772714T patent/PT4211231T/pt unknown
- 2021-09-10 CA CA3193128A patent/CA3193128A1/en active Pending
- 2021-09-10 WO PCT/EP2021/074964 patent/WO2022053630A1/en not_active Ceased
- 2021-09-10 FI FIEP21777271.4T patent/FI4211231T3/fi active
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015006743A1 (en) | 2013-07-12 | 2015-01-15 | The Children's Hospital Of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| WO2019193119A1 (en) | 2018-04-05 | 2019-10-10 | Genethon | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
| WO2019207132A1 (en) | 2018-04-27 | 2019-10-31 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
Also Published As
| Publication number | Publication date |
|---|---|
| EP4211231B1 (en) | 2024-06-26 |
| JP2023544683A (ja) | 2023-10-25 |
| ES2984259T3 (es) | 2024-10-29 |
| CN116096431A (zh) | 2023-05-09 |
| DK4211231T3 (da) | 2024-07-08 |
| EP4421179A2 (en) | 2024-08-28 |
| US20240209030A1 (en) | 2024-06-27 |
| EP4211231A1 (en) | 2023-07-19 |
| FI4211231T3 (fi) | 2024-07-17 |
| WO2022053630A1 (en) | 2022-03-17 |
| EP4421179A3 (en) | 2024-12-18 |
| BR112023004176A2 (pt) | 2023-04-11 |
| PT4211231T (pt) | 2024-07-24 |
| PL4211231T3 (pl) | 2024-11-04 |
| CA3193128A1 (en) | 2022-03-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7834725B2 (ja) | ペプチド改変されたａａｖカプシド | |
| JP7374119B2 (ja) | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 | |
| WO2021219762A1 (en) | Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders | |
| US20230242905A1 (en) | Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping | |
| EP4536686B1 (en) | Peptide-modified aav capsid with enhanced muscle transduction efficiency | |
| BR112020021903A2 (pt) | terapia genética para degeneração do snc | |
| HK40091751A (en) | Peptide-modified aav capsid | |
| US20230272433A1 (en) | Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof | |
| BR112023004176B1 (pt) | Proteína de capsídeo de aav, polinucleotídeo, plasmídeo recombinante, partícula de vetor de aav, seu uso para tratamento de doenças musculares e composição farmacêutica | |
| JP2023532806A (ja) | 新規の筋肉特異的プロモーター | |
| WO2026041758A1 (en) | Modified aav capsid with enhanced transduction efficiency | |
| HK40118571A (zh) | 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A529 | Written submission of copy of amendment under article 34 pct |
Free format text: JAPANESE INTERMEDIATE CODE: A529 Effective date: 20230426 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20240909 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20240909 |
|
| A871 | Explanation of circumstances concerning accelerated examination |
Free format text: JAPANESE INTERMEDIATE CODE: A871 Effective date: 20240909 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20241209 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20250307 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250509 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20250527 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250806 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20250819 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20251119 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A821 Effective date: 20251120 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20260210 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20260311 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 7834725 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |