ES2960880T3 - Terapia génica mediada por AAV que restaura el gen de la otoferlina - Google Patents

Terapia génica mediada por AAV que restaura el gen de la otoferlina Download PDF

Info

Publication number
ES2960880T3
ES2960880T3 ES20701951T ES20701951T ES2960880T3 ES 2960880 T3 ES2960880 T3 ES 2960880T3 ES 20701951 T ES20701951 T ES 20701951T ES 20701951 T ES20701951 T ES 20701951T ES 2960880 T3 ES2960880 T3 ES 2960880T3
Authority
ES
Spain
Prior art keywords
otoferlin
seq
patients
mice
dfnb9
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
ES20701951T
Other languages
English (en)
Spanish (es)
Inventor
Saaid Safieddine
Christine Petit
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.)
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Institut Pasteur
Original Assignee
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
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=ES2960880(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Centre National de la Recherche Scientifique CNRS, Institut National de la Sante et de la Recherche Medicale INSERM, Institut Pasteur filed Critical Centre National de la Recherche Scientifique CNRS
Application granted granted Critical
Publication of ES2960880T3 publication Critical patent/ES2960880T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A01K2217/056Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to mutation of coding region of the transgene (dominant negative)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/20Animal model comprising regulated expression system
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14144Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated 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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Marine Sciences & Fisheries (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ES20701951T 2019-01-18 2020-01-20 Terapia génica mediada por AAV que restaura el gen de la otoferlina Active ES2960880T3 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ES2960880T3 true ES2960880T3 (es) 2024-03-07

Family

ID=65351986

Family Applications (1)

Application Number Title Priority Date Filing Date
ES20701951T Active ES2960880T3 (es) 2019-01-18 2020-01-20 Terapia génica mediada por AAV que restaura el gen de la otoferlina

Country Status (22)

Country Link
US (1) US20220125875A1 (https=)
EP (2) EP4295913A3 (https=)
JP (2) JP7595013B2 (https=)
KR (2) KR20260046255A (https=)
CN (1) CN113924109A (https=)
AU (2) AU2020208933B2 (https=)
BR (1) BR112021013996A2 (https=)
CA (1) CA3126884A1 (https=)
DK (1) DK3911354T5 (https=)
ES (1) ES2960880T3 (https=)
FI (1) FI3911354T3 (https=)
HR (1) HRP20231296T1 (https=)
HU (1) HUE063458T2 (https=)
IL (1) IL284742B2 (https=)
LT (1) LT3911354T (https=)
MX (1) MX2021008622A (https=)
PL (1) PL3911354T3 (https=)
PT (1) PT3911354T (https=)
RS (1) RS64794B1 (https=)
SI (1) SI3911354T1 (https=)
SM (1) SMT202300359T1 (https=)
WO (1) WO2020148458A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
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
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
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
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
AU2021376225A1 (en) * 2020-11-06 2023-06-22 Eli Lilly And Company Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
CN117295529A (zh) * 2021-02-19 2023-12-26 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方法
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
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
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
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

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
WO2003059396A1 (en) 2002-01-11 2003-07-24 Sergei Zolotukhin 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
KR102234672B1 (ko) 2013-05-21 2021-04-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US10751385B2 (en) * 2015-02-20 2020-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
SG11201804814YA (en) * 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP2019530737A (ja) 2016-08-23 2019-10-24 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性聴力障害を治療するための組成物および方法
AU2018261769B2 (en) * 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
US11660353B2 (en) * 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

Also Published As

Publication number Publication date
EP4295913A3 (en) 2024-03-06
EP3911354B8 (en) 2024-04-03
KR102946735B1 (ko) 2026-04-03
AU2025271467A1 (en) 2026-01-08
JP2025032155A (ja) 2025-03-11
LT3911354T (lt) 2023-11-27
MX2021008622A (es) 2021-12-15
PL3911354T3 (pl) 2024-03-11
WO2020148458A1 (en) 2020-07-23
SI3911354T1 (sl) 2023-12-29
EP3911354A1 (en) 2021-11-24
JP2022522996A (ja) 2022-04-21
HRP20231296T1 (hr) 2024-02-16
EP4295913A2 (en) 2023-12-27
IL284742B2 (en) 2024-06-01
KR20260046255A (ko) 2026-04-06
FI3911354T3 (fi) 2023-10-17
DK3911354T5 (da) 2024-08-05
EP3911354B1 (en) 2023-07-19
CA3126884A1 (en) 2020-07-23
SMT202300359T1 (it) 2023-11-13
KR20210118112A (ko) 2021-09-29
IL284742A (en) 2021-08-31
BR112021013996A2 (pt) 2021-10-26
AU2020208933A8 (en) 2021-08-26
AU2020208933A1 (en) 2021-07-29
US20220125875A1 (en) 2022-04-28
DK3911354T3 (da) 2023-10-16
JP7595013B2 (ja) 2024-12-05
IL284742B1 (en) 2024-02-01
AU2020208933B2 (en) 2025-08-28
CN113924109A (zh) 2022-01-11
HUE063458T2 (hu) 2024-01-28
PT3911354T (pt) 2023-10-24
RS64794B1 (sr) 2023-11-30

Similar Documents

Publication Publication Date Title
ES2960880T3 (es) Terapia génica mediada por AAV que restaura el gen de la otoferlina
ES2890813T3 (es) Tratamiento de AMD mediante el uso de AAV SFLT-1
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
US20130095071A1 (en) Method of treating or preventing hearing loss
ES2982270T3 (es) Constructos y procedimientos de terapia génica para el tratamiento de la pérdida de audición
US20220096658A1 (en) Adeno-associated viruses and their uses for inner ear therapy
US20220348957A1 (en) Adeno-associated viruses and their uses for inner ear therapy
US20250295716A1 (en) Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof
US11827680B2 (en) Compounds and methods of treating usher syndrome III
HK40105471A (en) Aav-mediated gene therapy restoring the otoferlin gene
US20240050520A1 (en) Gene therapy for treating usher syndrome
US20110171202A1 (en) Method of treating hearing loss using xiap
ES2901613T3 (es) Lebecetina, una lectina de tipo C, como inhibidor de la neovascularización
Class et al. Patent application title: METHOD OF TREATING OR PREVENTING HEARING LOSS Inventors: Manohar Bance (Halifax, CA) Manohar Bance (Halifax, CA) George Robertson (Halifax, CA) Jian Wang (Halifax, CA) Assignees: Audigen Inc.