JP2022506613A - 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 - Google Patents
内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 Download PDFInfo
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- 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
- 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
- A61P27/16—Otologicals
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- 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
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- 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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- 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
- C12N15/65—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression using markers
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03001—Alkaline phosphatase (3.1.3.1)
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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
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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- Zoology (AREA)
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- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (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 |
|---|---|---|---|
| JP2024048210A JP2024086735A (ja) | 2018-11-07 | 2024-03-25 | 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862756953P | 2018-11-07 | 2018-11-07 | |
| US62/756,953 | 2018-11-07 | ||
| US201962888093P | 2019-08-16 | 2019-08-16 | |
| US62/888,093 | 2019-08-16 | ||
| PCT/US2019/060328 WO2020097372A1 (en) | 2018-11-07 | 2019-11-07 | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024048210A Division JP2024086735A (ja) | 2018-11-07 | 2024-03-25 | 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022506613A true JP2022506613A (ja) | 2022-01-17 |
| JPWO2020097372A5 JPWO2020097372A5 (https=) | 2023-07-07 |
| JP2022506613A5 JP2022506613A5 (https=) | 2023-07-07 |
Family
ID=70611216
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021524060A Pending JP2022506613A (ja) | 2018-11-07 | 2019-11-07 | 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 |
| JP2024048210A Pending JP2024086735A (ja) | 2018-11-07 | 2024-03-25 | 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024048210A Pending JP2024086735A (ja) | 2018-11-07 | 2024-03-25 | 内耳の有毛細胞及び支持細胞における遺伝子欠陥/発現タンパク質を修正するためのアデノ随伴ウイルスベクターの使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20230056182A1 (https=) |
| EP (2) | EP4063510A1 (https=) |
| JP (2) | JP2022506613A (https=) |
| CN (2) | CN118497276A (https=) |
| AU (1) | AU2019377115A1 (https=) |
| CA (1) | CA3119065A1 (https=) |
| WO (1) | WO2020097372A1 (https=) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| MX2020006435A (es) | 2017-12-19 | 2021-02-09 | Akouos Inc | Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno. |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN119410636A (zh) | 2018-04-27 | 2025-02-11 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| AU2020218547A1 (en) | 2019-02-08 | 2021-08-26 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS |
| CA3159549A1 (en) | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| EP4164695A4 (en) * | 2020-05-13 | 2024-06-05 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS |
| US20240016955A1 (en) * | 2020-09-14 | 2024-01-18 | President And Fellows Of Harvard College | Dual-aav vector delivery of pcdh15 and uses thereof |
| JP2023541443A (ja) * | 2020-09-14 | 2023-10-02 | プレジデント アンド フェローズ オブ ハーバード カレッジ | GJB2をコードする組換えアデノ随伴ウイルス(rAAV)およびその使用。 |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| AU2021376225A1 (en) * | 2020-11-06 | 2023-06-22 | Eli Lilly And Company | Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss |
| IL303317A (en) * | 2020-12-01 | 2023-07-01 | Akouos Inc | ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA |
| MX2023007800A (es) | 2020-12-29 | 2023-07-11 | Akouos Inc | Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1. |
| US20220267795A1 (en) * | 2021-02-24 | 2022-08-25 | Kinase Pharma Inc. | Compositions and methods for regulating production of an angiogensis inhibitor |
| EP4473011A1 (en) * | 2022-02-02 | 2024-12-11 | Akouos, Inc. | Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms |
| KR20250080886A (ko) * | 2022-09-30 | 2025-06-05 | 데시벨 테라퓨틱스, 인크. | Slc26a4 조절 요소 및 이의 용도 |
| WO2025179016A1 (en) * | 2024-02-20 | 2025-08-28 | Skylark Bio, Inc. | Recombinant adeno associated virus and uses thereof |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001501819A (ja) * | 1996-09-25 | 2001-02-13 | セル・ジェネシス・インコーポレイテッド | アデノ随伴ウィルスベクターの非侵襲性投与 |
| JP2002516345A (ja) * | 1998-05-27 | 2002-06-04 | セル ジェネシス インコーポレイテッド | 第viii因子活性のアデノ随伴ウイルスベクター媒介性発現 |
| JP2016501023A (ja) * | 2012-12-04 | 2016-01-18 | メルク・シャープ・アンド・ドーム・コーポレーションM | 条件付き複製ウイルスベクター |
| WO2017100791A1 (en) * | 2015-12-11 | 2017-06-15 | Massachusetts Eye And Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011075838A1 (en) * | 2009-12-21 | 2011-06-30 | Audigen Inc. | Method of treating or preventing hearing loss |
| CA2909733C (en) * | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| TWI813532B (zh) * | 2015-06-18 | 2023-09-01 | 美商博得學院股份有限公司 | 降低脱靶效應的crispr酶突變 |
| WO2017136764A1 (en) * | 2016-02-05 | 2017-08-10 | The General Hospital Corporation | Hybrid system for efficient gene delivery to cells of the inner ear |
| WO2017181021A1 (en) * | 2016-04-15 | 2017-10-19 | Regenxbio Inc. | Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab |
| JP2019530737A (ja) * | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| KR102746901B1 (ko) * | 2017-03-03 | 2024-12-26 | 옵시디안 테라퓨틱스, 인크. | Cd19 조성물 및 면역요법을 위한 방법 |
-
2019
- 2019-11-07 CN CN202410531995.8A patent/CN118497276A/zh active Pending
- 2019-11-07 CN CN201980082439.4A patent/CN113853437A/zh active Pending
- 2019-11-07 JP JP2021524060A patent/JP2022506613A/ja active Pending
- 2019-11-07 WO PCT/US2019/060328 patent/WO2020097372A1/en not_active Ceased
- 2019-11-07 EP EP22155160.9A patent/EP4063510A1/en not_active Withdrawn
- 2019-11-07 EP EP19881503.7A patent/EP3880826A4/en not_active Withdrawn
- 2019-11-07 US US17/291,931 patent/US20230056182A1/en not_active Abandoned
- 2019-11-07 CA CA3119065A patent/CA3119065A1/en active Pending
- 2019-11-07 AU AU2019377115A patent/AU2019377115A1/en active Pending
-
2024
- 2024-03-25 JP JP2024048210A patent/JP2024086735A/ja active Pending
- 2024-08-15 US US18/806,641 patent/US20250041457A1/en active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2001501819A (ja) * | 1996-09-25 | 2001-02-13 | セル・ジェネシス・インコーポレイテッド | アデノ随伴ウィルスベクターの非侵襲性投与 |
| JP2002516345A (ja) * | 1998-05-27 | 2002-06-04 | セル ジェネシス インコーポレイテッド | 第viii因子活性のアデノ随伴ウイルスベクター媒介性発現 |
| JP2016501023A (ja) * | 2012-12-04 | 2016-01-18 | メルク・シャープ・アンド・ドーム・コーポレーションM | 条件付き複製ウイルスベクター |
| WO2017100791A1 (en) * | 2015-12-11 | 2017-06-15 | Massachusetts Eye And Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Non-Patent Citations (3)
| Title |
|---|
| INVEST OPHTHALMOL VIS SCI., vol. 59, JPN6023039456, October 2018 (2018-10-01), pages 4909 - 4920, ISSN: 0005327310 * |
| MOL THER METHODS CLIN DEV., vol. 11, JPN6023039455, 4 September 2018 (2018-09-04), pages 29 - 39, ISSN: 0005327309 * |
| 実験医学, vol. 12, no. 15, JPN6023039457, 1994, pages 41 - 46, ISSN: 0005327311 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020097372A8 (en) | 2021-04-29 |
| US20230056182A1 (en) | 2023-02-23 |
| US20250041457A1 (en) | 2025-02-06 |
| EP4063510A1 (en) | 2022-09-28 |
| JP2024086735A (ja) | 2024-06-28 |
| CN113853437A (zh) | 2021-12-28 |
| EP3880826A1 (en) | 2021-09-22 |
| AU2019377115A1 (en) | 2021-05-20 |
| EP3880826A4 (en) | 2022-08-24 |
| CN118497276A (zh) | 2024-08-16 |
| CA3119065A1 (en) | 2020-05-14 |
| WO2020097372A1 (en) | 2020-05-14 |
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