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 PDF

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JP2022506613A
JP2022506613A JP2021524060A JP2021524060A JP2022506613A JP 2022506613 A JP2022506613 A JP 2022506613A JP 2021524060 A JP2021524060 A JP 2021524060A JP 2021524060 A JP2021524060 A JP 2021524060A JP 2022506613 A JP2022506613 A JP 2022506613A
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amino acid
acid substitutions
amino acids
sequence
target protein
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エマニュエル ジェイ. サイモンズ,
ロバート ング,
ダニエル レンズ,
ミシェル バレロ,
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アコーオス インコーポレイテッド
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JP2021524060A 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 Pending JP2022506613A (ja)

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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)

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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

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JPWO2020097372A5 JPWO2020097372A5 (https=) 2023-07-07
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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

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US (2) US20230056182A1 (https=)
EP (2) EP4063510A1 (https=)
JP (2) JP2022506613A (https=)
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AU (1) AU2019377115A1 (https=)
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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
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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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