MX2022005236A - Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. - Google Patents
Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.Info
- Publication number
- MX2022005236A MX2022005236A MX2022005236A MX2022005236A MX2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A MX 2022005236 A MX2022005236 A MX 2022005236A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- hearing loss
- sensorineural hearing
- otoferlin
- methods
- Prior art date
Links
- 102000016774 Otoferlin Human genes 0.000 title abstract 5
- 108050006335 Otoferlin Proteins 0.000 title abstract 5
- 239000013598 vector Substances 0.000 title abstract 4
- 206010011891 Deafness neurosensory Diseases 0.000 title abstract 3
- 208000009966 Sensorineural Hearing Loss Diseases 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 231100000879 sensorineural hearing loss Toxicity 0.000 title abstract 3
- 208000023573 sensorineural hearing loss disease Diseases 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 230000009977 dual effect Effects 0.000 title 1
- 102000001708 Protein Isoforms Human genes 0.000 abstract 2
- 108010029485 Protein Isoforms Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000040430 polynucleotide Human genes 0.000 abstract 2
- 108091033319 polynucleotide Proteins 0.000 abstract 2
- 239000002157 polynucleotide Substances 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 101150006256 Otof gene Proteins 0.000 abstract 1
- 208000002982 auditory neuropathy Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
Classifications
-
- 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
- C07K14/705—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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
-
- 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
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Luminescent Compositions (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962928290P | 2019-10-30 | 2019-10-30 | |
| US202062965776P | 2020-01-24 | 2020-01-24 | |
| US202062971504P | 2020-02-07 | 2020-02-07 | |
| US202063023058P | 2020-05-11 | 2020-05-11 | |
| PCT/US2020/058265 WO2021087296A1 (en) | 2019-10-30 | 2020-10-30 | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2022005236A true MX2022005236A (es) | 2022-06-08 |
Family
ID=75716489
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022005236A MX2022005236A (es) | 2019-10-30 | 2020-10-30 | Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12589168B2 (OSRAM) |
| EP (1) | EP4051698A4 (OSRAM) |
| JP (2) | JP7665609B2 (OSRAM) |
| KR (1) | KR20220130093A (OSRAM) |
| BR (1) | BR112022008214A2 (OSRAM) |
| CA (1) | CA3159549A1 (OSRAM) |
| IL (1) | IL292620A (OSRAM) |
| MX (1) | MX2022005236A (OSRAM) |
| TW (1) | TWI896570B (OSRAM) |
| WO (1) | WO2021087296A1 (OSRAM) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| KR20240053630A (ko) * | 2021-09-10 | 2024-04-24 | 앵스띠뛰 파스퇴르 | 오토페를린의 이소형 5를 인코딩하는 이중 재조합 aav8 벡터 시스템 및 이의 용도 |
| WO2023150689A2 (en) * | 2022-02-04 | 2023-08-10 | Decible Therapeutics, Inc. | Gjb2 regulatory elements and uses thereof |
| 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 |
| IL322655A (en) * | 2023-02-17 | 2025-10-01 | Decibel Therapeutics Inc | Method and compositions for administering a dual autopralin vector system |
| WO2025221253A1 (en) * | 2024-04-17 | 2025-10-23 | Decibel Therapeutics, Inc. | Methods for treating sensorineural hearing loss using otoferlin dual vector systems |
Family Cites Families (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9720465D0 (en) | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| CA2386546A1 (en) | 1999-10-07 | 2001-04-12 | University Of Iowa Research Foundation | Adeno-associated viruses and uses thereof |
| WO2001029243A1 (en) | 1999-10-15 | 2001-04-26 | Dalhousie University | Method and vector for producing and transferring trans-spliced peptides |
| AU4445701A (en) | 2000-03-24 | 2001-10-03 | Centre National De La Recherche Scientifique-Cnrs | Multiple human and mouse otoferlin isoforms |
| US20040072154A1 (en) | 2000-12-22 | 2004-04-15 | Morris David W. | Novel compositions and methods for cancer |
| WO2002082904A2 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| JP2003088388A (ja) | 2001-09-14 | 2003-03-25 | Herikkusu Kenkyusho:Kk | 新規な全長ｃＤＮＡ |
| 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 |
| US20070161031A1 (en) | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
| 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. |
| CN101990433B (zh) | 2008-02-07 | 2014-11-05 | 马萨诸塞眼科耳科诊所 | 提高Atoh1表达的化合物 |
| US20090215879A1 (en) * | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| 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 |
| KR102057540B1 (ko) * | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| JP6342886B2 (ja) | 2012-04-18 | 2018-06-13 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法 |
| US20150209406A1 (en) | 2012-09-07 | 2015-07-30 | Massachusetts Eye And Ear Infirmary | Methods and compositions for regenerating hair cells and/or supporting cells |
| CA2904164C (en) | 2013-03-11 | 2022-10-18 | Fondazione Telethon | Mir-204 and mir-211 and uses thereof |
| EP2986635B1 (en) | 2013-04-18 | 2018-10-03 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| 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 |
| JP2018531926A (ja) | 2015-09-17 | 2018-11-01 | コーダ バイオセラピューティクス， インコーポレイテッド | 神経学的障害を処置するための組成物および方法 |
| 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 |
| AU2018215785B2 (en) | 2017-02-06 | 2023-08-31 | Children's Medical Center Corporation | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| 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 |
| 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 |
| US20190345516A1 (en) * | 2018-03-12 | 2019-11-14 | Musc Foundation For Research Development | Methods for treating eye disease |
| AU2019237541B2 (en) | 2018-03-23 | 2025-02-13 | Editas Medicine, Inc. | CRISPR/Cas9-mediated exon-skipping approach for USH2A-associated Usher syndrome |
| 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 |
| WO2020093018A1 (en) * | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| 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 |
| MX2021008542A (es) | 2019-01-18 | 2021-11-12 | Voyager Therapeutics Inc | Métodos y sistemas para la producción de partículas de aav. |
| BR112021013996A2 (pt) | 2019-01-18 | 2021-10-26 | Institut Pasteur | Terapia gênica mediada por aav que recupera o gene otoferlina |
| 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 TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS |
| 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 |
| WO2022178298A1 (en) | 2021-02-19 | 2022-08-25 | Decibel Therapeutics, Inc. | Methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| IL322655A (en) | 2023-02-17 | 2025-10-01 | Decibel Therapeutics Inc | Method and compositions for administering a dual autopralin vector system |
-
2020
- 2020-10-30 JP JP2022525387A patent/JP7665609B2/ja active Active
- 2020-10-30 KR KR1020227018008A patent/KR20220130093A/ko active Pending
- 2020-10-30 CA CA3159549A patent/CA3159549A1/en active Pending
- 2020-10-30 TW TW109137935A patent/TWI896570B/zh active
- 2020-10-30 EP EP20883297.2A patent/EP4051698A4/en active Pending
- 2020-10-30 WO PCT/US2020/058265 patent/WO2021087296A1/en not_active Ceased
- 2020-10-30 IL IL292620A patent/IL292620A/en unknown
- 2020-10-30 MX MX2022005236A patent/MX2022005236A/es unknown
- 2020-10-30 BR BR112022008214A patent/BR112022008214A2/pt unknown
-
2022
- 2022-04-29 US US17/733,744 patent/US12589168B2/en active Active
-
2025
- 2025-04-09 JP JP2025064211A patent/JP2025106464A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7665609B2 (ja) | 2025-04-21 |
| CA3159549A1 (en) | 2021-05-06 |
| JP2025106464A (ja) | 2025-07-15 |
| TWI896570B (zh) | 2025-09-11 |
| BR112022008214A2 (pt) | 2022-07-12 |
| US20220265865A1 (en) | 2022-08-25 |
| WO2021087296A8 (en) | 2021-08-05 |
| IL292620A (en) | 2022-07-01 |
| KR20220130093A (ko) | 2022-09-26 |
| JP2022554300A (ja) | 2022-12-28 |
| EP4051698A1 (en) | 2022-09-07 |
| TW202130654A (zh) | 2021-08-16 |
| US12589168B2 (en) | 2026-03-31 |
| EP4051698A4 (en) | 2024-03-27 |
| WO2021087296A1 (en) | 2021-05-06 |
| AU2020375947A1 (en) | 2022-05-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| WO2021087296A8 (en) | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems | |
| WO2020163743A8 (en) | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems | |
| MX2025009545A (es) | Metodos y composiciones para la administracion de sistemas de vector dual otoferlin | |
| MX2023009714A (es) | Metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina. | |
| MY191030A (en) | Gdf15 fusion proteins and uses thereof | |
| PH12019500122A1 (en) | Bispecific antibody-like binding proteins specifically binding to cd3 and cd123 | |
| EA202091995A1 (ru) | Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека | |
| MX2019006045A (es) | Proteinas triespecificas dirigidas a psma y metodos de uso. | |
| MX2023001394A (es) | Genes del factor viii optimizados. | |
| WO2020093018A8 (en) | A codon optimized otoferlin aav dual vector gene therapy | |
| EP4219724A3 (en) | Compositions and methods for treating non-age-associated hearing impairment in a human subject | |
| PH12022550693A1 (en) | Multi-specific binding proteins for cancer treatment | |
| MY190926A (en) | Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders | |
| WO2018170150A3 (en) | Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins | |
| MX2021006253A (es) | Terapias génicas para enfermedad neurodegenerativa. | |
| WO2021127200A8 (en) | Ilt3-binding agents and methods of use thereof | |
| BR112022006842A2 (pt) | Construtos de igf2 variante | |
| MX2022010050A (es) | Composiciones y metodos para tratar deficiencia auditiva no asociada con la edad en un sujeto humano. | |
| WO2022150732A3 (en) | Chimeric receptor therapy | |
| ZA202104912B (en) | Rna encoding a protein | |
| WO2020150290A3 (en) | Methods and compositions for restoring stmn2 levels | |
| MX2023013053A (es) | Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de estereocilina. | |
| WO2020214809A3 (en) | Gene therapies for stargardt disease (abca4) | |
| MY205028A (en) | Protein for treatment of inflammatory diseases | |
| PH12020552222A1 (en) | Immunodominant proteins and fragments in multiple sclerosis |