JP2024537796A - 難聴を治療するための遺伝子療法送達組成物及び方法 - Google Patents

難聴を治療するための遺伝子療法送達組成物及び方法 Download PDF

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JP2024537796A
JP2024537796A JP2024519540A JP2024519540A JP2024537796A JP 2024537796 A JP2024537796 A JP 2024537796A JP 2024519540 A JP2024519540 A JP 2024519540A JP 2024519540 A JP2024519540 A JP 2024519540A JP 2024537796 A JP2024537796 A JP 2024537796A
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nucleic acid
construct
aspects
acid sequence
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ダイアン グリブル キャサリン
アール.レンズ ダニエル
ウン ロバート
チーアン ハオ
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アコーオス インコーポレイテッド
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • A61M25/0067Catheters; Hollow probes characterised by the distal end, e.g. tips
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    • A61M25/00Catheters; Hollow probes
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    • A61M2025/0042Microcatheters, cannula or the like having outside diameters around 1 mm or less
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    • A61M25/0067Catheters; Hollow probes characterised by the distal end, e.g. tips
    • A61M25/0082Catheter tip comprising a tool
    • A61M25/0084Catheter tip comprising a tool being one or more injection needles
    • A61M2025/0093Catheter tip comprising a tool being one or more injection needles wherein at least one needle is a microneedle
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JP2024519540A 2021-09-30 2022-09-30 難聴を治療するための遺伝子療法送達組成物及び方法 Pending JP2024537796A (ja)

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US202163251017P 2021-09-30 2021-09-30
US63/251,017 2021-09-30
PCT/US2022/077397 WO2023056452A1 (en) 2021-09-30 2022-09-30 Gene therapy delivery compositions and methods for treating hearing loss

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JP2024537796A true JP2024537796A (ja) 2024-10-16
JP2024537796A5 JP2024537796A5 (https=) 2025-12-09

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US (1) US20250222137A1 (https=)
EP (1) EP4409009A4 (https=)
JP (1) JP2024537796A (https=)
KR (1) KR20240099153A (https=)
CN (1) CN118339302A (https=)
AU (1) AU2022358579A1 (https=)
CA (1) CA3233522A1 (https=)
MX (1) MX2024003910A (https=)
WO (1) WO2023056452A1 (https=)

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Publication number Priority date Publication date Assignee Title
US20240382461A1 (en) * 2023-05-15 2024-11-21 Pharmazz, Inc. Pharmaceutical composition and method for prevention and treatment of hearing loss
CN119859633A (zh) * 2024-12-31 2025-04-22 中国水产科学研究院东海水产研究所 一种拟穴青蟹eyes absent-2基因编辑的方法及应用

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3762040A4 (en) * 2018-03-05 2022-01-05 Children's Medical Center Corporation COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS
US20210277417A1 (en) * 2018-06-25 2021-09-09 Akouos, Inc. Methods of treating clrn1-associated hearing loss and/or vision loss
WO2021091938A1 (en) * 2019-11-04 2021-05-14 Decibel Therapeutics, Inc. Cochlear outer hair cell promoters and uses thereof
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途

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AU2022358579A1 (en) 2024-04-11
EP4409009A1 (en) 2024-08-07
WO2023056452A1 (en) 2023-04-06
KR20240099153A (ko) 2024-06-28
CA3233522A1 (en) 2023-04-06
US20250222137A1 (en) 2025-07-10
MX2024003910A (es) 2024-06-28
CN118339302A (zh) 2024-07-12
EP4409009A4 (en) 2025-12-17

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