KR20230023641A - Gjb2-연관 난청을 치료하기 위한 조성물 및 방법 - Google Patents

Gjb2-연관 난청을 치료하기 위한 조성물 및 방법 Download PDF

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KR20230023641A
KR20230023641A KR1020227043071A KR20227043071A KR20230023641A KR 20230023641 A KR20230023641 A KR 20230023641A KR 1020227043071 A KR1020227043071 A KR 1020227043071A KR 20227043071 A KR20227043071 A KR 20227043071A KR 20230023641 A KR20230023641 A KR 20230023641A
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promoter
cells
seq
construct
nucleic acid
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Korean (ko)
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엠마뉴엘 존 시몬스
로버트 엔쥐
다니엘 알. 렌즈
하오 치앙
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아카우오스, 인크.
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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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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    • 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
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    • A61M25/00—Catheters; Hollow probes
    • A61M25/0067—Catheters; Hollow probes characterised by the distal end, e.g. tips
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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
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    • A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61M25/00—Catheters; Hollow probes
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    • A61M25/00—Catheters; Hollow probes
    • A61M25/0067—Catheters; Hollow probes characterised by the distal end, e.g. tips
    • A61M25/0082—Catheter tip comprising a tool
    • A61M25/0084—Catheter tip comprising a tool being one or more injection needles
    • A61M2025/0093—Catheter tip comprising a tool being one or more injection needles wherein at least one needle is a microneedle
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Heart & Thoracic Surgery (AREA)
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KR1020227043071A 2020-05-13 2021-05-13 Gjb2-연관 난청을 치료하기 위한 조성물 및 방법 Pending KR20230023641A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063024468P 2020-05-13 2020-05-13
US63/024,468 2020-05-13
US202163152835P 2021-02-23 2021-02-23
US63/152,835 2021-02-23
PCT/US2021/032354 WO2021231808A2 (en) 2020-05-13 2021-05-13 Compositions and methods for treating gjb2-associated hearing loss

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Publication Number Publication Date
KR20230023641A true KR20230023641A (ko) 2023-02-17

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KR1020227043071A Pending KR20230023641A (ko) 2020-05-13 2021-05-13 Gjb2-연관 난청을 치료하기 위한 조성물 및 방법

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US (1) US20230183743A1 (https=)
EP (1) EP4164695A4 (https=)
JP (1) JP2023526053A (https=)
KR (1) KR20230023641A (https=)
CN (1) CN116096900A (https=)
AU (1) AU2021270308A1 (https=)
CA (1) CA3178197A1 (https=)
CL (1) CL2022003147A1 (https=)
IL (1) IL298091A (https=)
MX (1) MX2022014085A (https=)
WO (1) WO2021231808A2 (https=)

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Publication number Priority date Publication date Assignee Title
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
TW202227476A (zh) * 2020-09-14 2022-07-16 哈佛大學校長及研究員協會 編碼GJB2之重組腺相關病毒(rAAV)及其用途
US20230340038A1 (en) * 2020-09-14 2023-10-26 President And Fellows Of Harvard College Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof
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
MX2023013394A (es) * 2021-05-13 2023-12-14 Akouos Inc Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva.
CN115651932A (zh) * 2021-12-10 2023-01-31 浙江理工大学 一种靶向消化道肿瘤双靶向溶瘤腺病毒的构建方法及其应用
MX2024009520A (es) * 2022-02-04 2024-09-11 Decibel Therapeutics Inc Elementos reguladores de gjb2 y usos de los mismos.
CN116836975A (zh) * 2022-03-25 2023-10-03 上海玮美基因科技有限责任公司 一种耳蜗和/或前庭细胞特异性启动子及其应用
CN119061007A (zh) * 2023-06-02 2024-12-03 苏州星奥拓维生物技术有限公司 耳蜗支持细胞基因治疗和再生的方法
CN117924458A (zh) * 2024-01-26 2024-04-26 嘉兴元圣生物科技有限公司 一种获取果蝠connexin30蛋白编码cDNA序列、氨基酸序列的方法及其序列的应用

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EP0173552B1 (en) * 1984-08-24 1991-10-09 The Upjohn Company Recombinant dna compounds and the expression of polypeptides such as tpa
WO2005009287A2 (en) * 2003-07-21 2005-02-03 University Of Maryland, Baltimore Drug delivery to the inner ear and methods of using same
US20150050354A1 (en) * 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
HRP20180487T1 (hr) * 2013-07-24 2018-05-04 Commissariat à l'énergie atomique et aux énergies alternatives Upotreba flekainida kao anti-koneksinskog sredstva i metoda za potenciranje učinaka psihotropnog lijeka
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2019200016A1 (en) * 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
EP4063510A1 (en) * 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
WO2021067448A1 (en) * 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss

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US20230183743A1 (en) 2023-06-15
WO2021231808A3 (en) 2021-12-23
EP4164695A4 (en) 2024-06-05
WO2021231808A2 (en) 2021-11-18
AU2021270308A1 (en) 2022-12-15
CL2022003147A1 (es) 2023-07-21
MX2022014085A (es) 2022-12-07
IL298091A (en) 2023-01-01
JP2023526053A (ja) 2023-06-20
CA3178197A1 (en) 2021-11-18
CN116096900A (zh) 2023-05-09
EP4164695A2 (en) 2023-04-19

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