JP7240675B2 - オトフェルリンを発現させるための組成物および方法 - Google Patents

オトフェルリンを発現させるための組成物および方法 Download PDF

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JP7240675B2
JP7240675B2 JP2019560646A JP2019560646A JP7240675B2 JP 7240675 B2 JP7240675 B2 JP 7240675B2 JP 2019560646 A JP2019560646 A JP 2019560646A JP 2019560646 A JP2019560646 A JP 2019560646A JP 7240675 B2 JP7240675 B2 JP 7240675B2
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sequence
otof
composition
aav
polynucleotide
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JP2020518268A (ja
JP2020518268A5 (OSRAM
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ボエ，サンフォード，エル．
デュカ，フランク
ハウスワース，ウイリアム，ダブリュー．
アキル，オマール
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University of California San Diego UCSD
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • 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
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • A—HUMAN NECESSITIES
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    • 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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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C—CHEMISTRY; METALLURGY
    • 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
    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
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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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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • 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)
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JP2019560646A 2017-05-05 2018-05-04 オトフェルリンを発現させるための組成物および方法 Active JP7240675B2 (ja)

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JP2023027073A JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

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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

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JP2023027073A Active JP7617600B2 (ja) 2017-05-05 2023-02-24 オトフェルリンを発現させるための組成物および方法
JP2024226066A Active JP7851563B2 (ja) 2017-05-05 2024-12-23 オトフェルリンを発現させるための組成物および方法

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US (2) US12359221B2 (OSRAM)
EP (1) EP3635100A4 (OSRAM)
JP (3) JP7240675B2 (OSRAM)
KR (2) KR102606810B1 (OSRAM)
CN (1) CN110892062A (OSRAM)
AU (2) AU2018261769B2 (OSRAM)
CA (1) CA3061955A1 (OSRAM)
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023070682A (ja) * 2017-05-05 2023-05-19 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
US12377169B2 (en) 2020-04-01 2025-08-05 University Of Florida Research Foundation, Incorporated Dual AAV-MYO7A vectors with improved safety for the treatment of USH1B

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
DE102018103924A1 (de) * 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
IL276764B2 (en) * 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
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
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
BR112021013996A2 (pt) * 2019-01-18 2021-10-26 Institut Pasteur Terapia gênica mediada por aav que recupera o gene otoferlina
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
JP7562544B2 (ja) 2019-02-08 2024-10-07 デシベル セラピューティクス インコーポレイテッド ミオシン１５プロモーター及びその使用
JP7665609B2 (ja) * 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
CN115066432B (zh) * 2019-10-30 2026-04-17 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的组合物和方法
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
CN112125969A (zh) * 2020-09-30 2020-12-25 东南大学 生物因子rimbp2在维持内耳毛细胞特性上的应用
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
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017049252A1 (en) 2015-09-17 2017-03-23 Switch Bio, Inc. Compositions and methods for treating neurological disorders

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4445701A (en) * 2000-03-24 2001-10-03 Centre National De La Recherche Scientifique-Cnrs Multiple human and mouse otoferlin isoforms
WO2005071092A1 (ja) 2004-01-22 2005-08-04 Dnavec Research Inc. サイトメガロウイルスエンハンサーおよびニワトリβ-アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖RNAウイルスベクターの製造方法
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
JP5244103B2 (ja) 2006-08-09 2013-07-24 ホームステッド クリニカル コーポレイション 器官特異的蛋白質およびその使用方法
US7892824B2 (en) 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010062905A2 (en) * 2008-11-26 2010-06-03 The Regents Of The University Of California Lateral ventricle cell compositions and use for treating neural degenerative diseases
KR20200047642A (ko) * 2010-04-23 2020-05-07 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
CA2904164C (en) 2013-03-11 2022-10-18 Fondazione Telethon Mir-204 and mir-211 and uses thereof
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11000597B2 (en) 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
WO2016131981A1 (en) * 2015-02-20 2016-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
CN116236591B (zh) 2015-12-11 2026-02-27 马萨诸塞眼科耳科诊所 用于将核酸递送至耳蜗和前庭细胞的材料和方法
EP3393522B1 (en) 2015-12-22 2021-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
WO2017216560A1 (en) 2016-06-15 2017-12-21 Oxford University Innovation Limited Dual overlapping adeno-associated viral vector system for expressing abc4a
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018162748A1 (en) 2017-03-10 2018-09-13 Genethon Treatment of glycogen storage disease iii
JP7240675B2 (ja) 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
IL276764B2 (en) 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
AU2019237541B2 (en) 2018-03-23 2025-02-13 Editas Medicine, Inc. CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
BR112021013996A2 (pt) 2019-01-18 2021-10-26 Institut Pasteur Terapia gênica mediada por aav que recupera o gene otoferlina
JP7665609B2 (ja) 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a vectors with improved safety for the treatment of ush1b

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017049252A1 (en) 2015-09-17 2017-03-23 Switch Bio, Inc. Compositions and methods for treating neurological disorders

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Arkasubhra Ghosh et al.，A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner，Molecular Therapy，2008年，Vol.16、No.1，p. 124-130
Arkasubhra Ghosh et al.，Efficient Transgene Reconstitution with Hybrid Dual AAV Vectors Carrying the Minimized Bridging Sequences，HUMAN GENE THERAPY，2011年，Vol.22、No.1，p. 77-83
Aurash Alemi et al.，AOS Research Grant: Restoration of Hearing in the Otoferlin Knockout Mouse using Viral Gene Therapy，PROGRAM and ABSTRACTS of the One Hundred Forty-Fifth Annual Meeting AMERICAN OTOLOGICAL SOCIETY, INC. April 2l-22, 2012，2012年，p. 68，https://www.americanotologicalsociety.org/assets/2012.pdf

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JP2023070682A (ja) * 2017-05-05 2023-05-19 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
JP7617600B2 (ja) 2017-05-05 2025-01-20 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
JP2025063057A (ja) * 2017-05-05 2025-04-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12359221B2 (en) 2017-05-05 2025-07-15 The Regents Of The University Of California Compositions and methods for expressing otoferlin
JP7851563B2 (ja) 2017-05-05 2026-04-27 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
US12377169B2 (en) 2020-04-01 2025-08-05 University Of Florida Research Foundation, Incorporated Dual AAV-MYO7A vectors with improved safety for the treatment of USH1B

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