JP7595013B2 - オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 - Google Patents

オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 Download PDF

Info

Publication number
JP7595013B2
JP7595013B2 JP2021541524A JP2021541524A JP7595013B2 JP 7595013 B2 JP7595013 B2 JP 7595013B2 JP 2021541524 A JP2021541524 A JP 2021541524A JP 2021541524 A JP2021541524 A JP 2021541524A JP 7595013 B2 JP7595013 B2 JP 7595013B2
Authority
JP
Japan
Prior art keywords
sequence
otoferlin
pharmaceutical composition
seq
otof
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2021541524A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022522996A (ja
JPWO2020148458A5 (enExample
Inventor
サーイド、サフィーディーヌ
クリスティーヌ、プチ
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Institut Pasteur
Original Assignee
Institut Pasteur
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=65351986&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP7595013(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Institut Pasteur filed Critical Institut Pasteur
Publication of JP2022522996A publication Critical patent/JP2022522996A/ja
Publication of JPWO2020148458A5 publication Critical patent/JPWO2020148458A5/ja
Priority to JP2024204975A priority Critical patent/JP2025032155A/ja
Application granted granted Critical
Publication of JP7595013B2 publication Critical patent/JP7595013B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A01K2217/056—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to mutation of coding region of the transgene (dominant negative)
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Environmental Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021541524A 2019-01-18 2020-01-20 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療 Active JP7595013B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024204975A JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19305071 2019-01-18
EP19305071.3 2019-01-18
PCT/EP2020/051283 WO2020148458A1 (en) 2019-01-18 2020-01-20 Aav-mediated gene therapy restoring the otoferlin gene

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024204975A Division JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療

Publications (3)

Publication Number Publication Date
JP2022522996A JP2022522996A (ja) 2022-04-21
JPWO2020148458A5 JPWO2020148458A5 (enExample) 2024-05-10
JP7595013B2 true JP7595013B2 (ja) 2024-12-05

Family

ID=65351986

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021541524A Active JP7595013B2 (ja) 2019-01-18 2020-01-20 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療
JP2024204975A Pending JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024204975A Pending JP2025032155A (ja) 2019-01-18 2024-11-25 オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療

Country Status (22)

Country Link
US (1) US20220125875A1 (enExample)
EP (2) EP4295913A3 (enExample)
JP (2) JP7595013B2 (enExample)
KR (1) KR20210118112A (enExample)
CN (1) CN113924109A (enExample)
AU (1) AU2020208933B2 (enExample)
BR (1) BR112021013996A2 (enExample)
CA (1) CA3126884A1 (enExample)
DK (1) DK3911354T5 (enExample)
ES (1) ES2960880T3 (enExample)
FI (1) FI3911354T3 (enExample)
HR (1) HRP20231296T1 (enExample)
HU (1) HUE063458T2 (enExample)
IL (1) IL284742B2 (enExample)
LT (1) LT3911354T (enExample)
MX (1) MX2021008622A (enExample)
PL (1) PL3911354T3 (enExample)
PT (1) PT3911354T (enExample)
RS (1) RS64794B1 (enExample)
SI (1) SI3911354T1 (enExample)
SM (1) SMT202300359T1 (enExample)
WO (1) WO2020148458A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7331011B2 (ja) 2018-04-27 2023-08-22 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
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
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
WO2021202817A1 (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
WO2022099007A1 (en) * 2020-11-06 2022-05-12 Applied Genetic Technologies Corporation Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
KR20240004253A (ko) * 2021-02-19 2024-01-11 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 방법
MX2024003052A (es) * 2021-09-10 2024-05-14 Pasteur Institut Sistema de vectores aav8 recombinante dual que codifica la isoforma 5 de otoferlina y sus usos.
EP4527933A4 (en) * 2022-05-17 2025-12-24 Eye & Ent Hospital Of Fudan Univ DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE
CN117106824B (zh) * 2022-05-17 2025-09-02 上海佑音医药生物科技有限公司 一种治疗听力损伤的双载体系统及其应用
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
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520311A (ja) 2013-05-21 2016-07-14 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2018204734A1 (en) 2017-05-05 2018-11-08 University Of Florida Research Foundation, Incorporated Compositions and methods for expressing otoferlin
JP2018536420A (ja) 2015-12-11 2018-12-13 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
AU2003209226A1 (en) 2002-01-11 2003-07-30 Michael Tennant Adiponectin gene therapy
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
WO2011075838A1 (en) 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2019165292A1 (en) * 2018-02-22 2019-08-29 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11660353B2 (en) * 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
BR112022008214A2 (pt) 2019-10-30 2022-07-12 Decibel Therapeutics Inc Composições e métodos para tratar perda auditiva neurossensorial usando sistemas de vetor duplo de otoferlina

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520311A (ja) 2013-05-21 2016-07-14 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
JP2018536420A (ja) 2015-12-11 2018-12-13 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法
WO2018039375A1 (en) 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2018204734A1 (en) 2017-05-05 2018-11-08 University Of Florida Research Foundation, Incorporated Compositions and methods for expressing otoferlin

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
EMBO Molecular Medicine，Vol.11，e9396 (pp.1-13)，Published online 2018.12.03
Hearing Research，2016年，Vol.335，pp.53-63
Molecular Therapy，2011年，Vol.19, No.2，pp.293-301

Also Published As

Publication number Publication date
JP2022522996A (ja) 2022-04-21
SMT202300359T1 (it) 2023-11-13
RS64794B1 (sr) 2023-11-30
WO2020148458A1 (en) 2020-07-23
IL284742B2 (en) 2024-06-01
AU2020208933A8 (en) 2021-08-26
EP3911354A1 (en) 2021-11-24
LT3911354T (lt) 2023-11-27
ES2960880T3 (es) 2024-03-07
CN113924109A (zh) 2022-01-11
EP3911354B1 (en) 2023-07-19
PT3911354T (pt) 2023-10-24
IL284742B1 (en) 2024-02-01
HRP20231296T1 (hr) 2024-02-16
HUE063458T2 (hu) 2024-01-28
PL3911354T3 (pl) 2024-03-11
FI3911354T3 (fi) 2023-10-17
JP2025032155A (ja) 2025-03-11
IL284742A (en) 2021-08-31
DK3911354T5 (da) 2024-08-05
DK3911354T3 (da) 2023-10-16
KR20210118112A (ko) 2021-09-29
EP3911354B8 (en) 2024-04-03
SI3911354T1 (sl) 2023-12-29
CA3126884A1 (en) 2020-07-23
MX2021008622A (es) 2021-12-15
AU2020208933B2 (en) 2025-08-28
AU2020208933A1 (en) 2021-07-29
US20220125875A1 (en) 2022-04-28
BR112021013996A2 (pt) 2021-10-26
EP4295913A2 (en) 2023-12-27
EP4295913A3 (en) 2024-03-06

Similar Documents

Publication Publication Date Title
JP7595013B2 (ja) オトフェリン遺伝子を回復させるａａｖ媒介遺伝子治療
Ong et al. Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease: Ong et al.
Lipinski et al. Clinical applications of retinal gene therapy
JP6453307B2 (ja) デュアルａａｖベクターによる大型遺伝子の効果的送達
JP7430652B2 (ja) 蝸牛および前庭細胞に核酸を送達するための組成物および方法
KR20180043373A (ko) 색소성망막염의 치료
US20220378945A1 (en) Gene therapy targeting cochlear cells
US20250295716A1 (en) Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
US20240190929A1 (en) Compositions and methods of treating usher syndrome iii
US20220096658A1 (en) Adeno-associated viruses and their uses for inner ear therapy
CN117305367A (zh) 一种表达全长耳畸蛋白的双aav载体系统及其应用
US20220348957A1 (en) Adeno-associated viruses and their uses for inner ear therapy
HK40105471A (en) Aav-mediated gene therapy restoring the otoferlin gene
CN113795279B (zh) 靶向akt通路的神经保护性基因疗法
US20240050520A1 (en) Gene therapy for treating usher syndrome
CN118215508A (zh) 编码耳畸蛋白同工型5的双重组aav8载体系统及其用途
WO2025162267A1 (zh) 表达myo7a蛋白的双载体系统及其用途
WO2025157179A1 (zh) 表达strc蛋白的双载体系统及其用途
EA046019B1 (ru) Композиции и способы лечения нарушений сетчатки

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20221121

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20231025

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231027

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240115

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240304

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20240425

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240709

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241008

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20241025

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20241125

R150 Certificate of patent or registration of utility model

Ref document number: 7595013

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150