KR20230167138A - 오토펄린을 발현시키기 위한 조성물 및 방법 - Google Patents

오토펄린을 발현시키기 위한 조성물 및 방법 Download PDF

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KR20230167138A
KR20230167138A KR1020237040309A KR20237040309A KR20230167138A KR 20230167138 A KR20230167138 A KR 20230167138A KR 1020237040309 A KR1020237040309 A KR 1020237040309A KR 20237040309 A KR20237040309 A KR 20237040309A KR 20230167138 A KR20230167138 A KR 20230167138A
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샌포드 엘. 보예
프랭크 다이카
윌리엄 더블유. 하우스워쓰
오마르 아킬
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유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드
더 리전트 오브 더 유니버시티 오브 캘리포니아
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Application filed by 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드, 더 리전트 오브 더 유니버시티 오브 캘리포니아 filed Critical 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드
Publication of KR20230167138A publication Critical patent/KR20230167138A/ko
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07—ORGANIC CHEMISTRY
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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
    • C07K14/46—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
    • C—CHEMISTRY; METALLURGY
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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
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    • 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
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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
KR1020237040309A 2017-05-05 2018-05-04 오토펄린을 발현시키기 위한 조성물 및 방법 Ceased KR20230167138A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762502462P 2017-05-05 2017-05-05
US62/502,462 2017-05-05
PCT/US2018/031009 WO2018204734A1 (en) 2017-05-05 2018-05-04 Compositions and methods for expressing otoferlin
KR1020197035845A KR102606810B1 (ko) 2017-05-05 2018-05-04 오토펄린을 발현시키기 위한 조성물 및 방법

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KR1020197035845A Active KR102606810B1 (ko) 2017-05-05 2018-05-04 오토펄린을 발현시키기 위한 조성물 및 방법

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US (2) US12359221B2 (https=)
EP (1) EP3635100A4 (https=)
JP (2) JP7240675B2 (https=)
KR (2) KR20230167138A (https=)
CN (1) CN110892062A (https=)
AU (2) AU2018261769B2 (https=)
CA (1) CA3061955A1 (https=)
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AU2018261769B2 (en) * 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
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CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
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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
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B

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EP3635100A4 (en) 2021-03-24
WO2018204734A8 (en) 2018-12-06
KR20200003881A (ko) 2020-01-10
MX2019013151A (es) 2020-02-05
US20200157573A1 (en) 2020-05-21
CA3061955A1 (en) 2018-11-08
AU2018261769B2 (en) 2024-03-14
KR102606810B1 (ko) 2023-11-27
AU2024203084A1 (en) 2024-05-30
AU2018261769A1 (en) 2019-11-21
JP2025063057A (ja) 2025-04-15
AU2024203084B2 (en) 2026-04-02
WO2018204734A1 (en) 2018-11-08
CN110892062A (zh) 2020-03-17
JP2023070682A (ja) 2023-05-19
JP7240675B2 (ja) 2023-03-16
US12359221B2 (en) 2025-07-15
US20250367325A1 (en) 2025-12-04
EP3635100A1 (en) 2020-04-15
JP7617600B2 (ja) 2025-01-20
JP2020518268A (ja) 2020-06-25
MX2023013435A (es) 2023-12-12

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