SG11201804814YA - Materials and methods for delivering nucleic acids to cochlear and vestibular cells - Google Patents
Materials and methods for delivering nucleic acids to cochlear and vestibular cellsInfo
- Publication number
- SG11201804814YA SG11201804814YA SG11201804814YA SG11201804814YA SG11201804814YA SG 11201804814Y A SG11201804814Y A SG 11201804814YA SG 11201804814Y A SG11201804814Y A SG 11201804814YA SG 11201804814Y A SG11201804814Y A SG 11201804814YA SG 11201804814Y A SG11201804814Y A SG 11201804814YA
- Authority
- SG
- Singapore
- Prior art keywords
- massachusetts
- international
- street
- cochlear
- nucleic acids
- Prior art date
Links
- 239000000463 material Substances 0.000 title abstract 3
- 150000007523 nucleic acids Chemical class 0.000 title abstract 3
- 102000039446 nucleic acids Human genes 0.000 title abstract 3
- 108020004707 nucleic acids Proteins 0.000 title abstract 3
- 230000001720 vestibular Effects 0.000 title abstract 3
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 102220240796 rs553605556 Human genes 0.000 abstract 1
Classifications
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- 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
- 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
- A61K38/18—Growth factors; Growth regulators
- A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0046—Ear
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
- A61N1/00—Electrotherapy; Circuits therefor
- A61N1/18—Applying electric currents by contact electrodes
- A61N1/32—Applying electric currents by contact electrodes alternating or intermittent currents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P27/02—Ophthalmic agents
-
- 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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
- A61K2039/53—DNA (RNA) vaccination
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Zoology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Mycology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Physics & Mathematics (AREA)
- Radiology & Medical Imaging (AREA)
- Dermatology (AREA)
- Plant Pathology (AREA)
- Ophthalmology & Optometry (AREA)
- Psychology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266477P | 2015-12-11 | 2015-12-11 | |
| US201562266462P | 2015-12-11 | 2015-12-11 | |
| PCT/US2016/066225 WO2017100791A1 (en) | 2015-12-11 | 2016-12-12 | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201804814YA true SG11201804814YA (en) | 2018-07-30 |
Family
ID=59013436
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201804814YA SG11201804814YA (en) | 2015-12-11 | 2016-12-12 | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| SG10201913266UA SG10201913266UA (en) | 2015-12-11 | 2016-12-12 | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201913266UA SG10201913266UA (en) | 2015-12-11 | 2016-12-12 | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11167042B2 (https=) |
| EP (2) | EP3386537B1 (https=) |
| JP (3) | JP6990182B2 (https=) |
| KR (1) | KR102794870B1 (https=) |
| CN (2) | CN116236591B (https=) |
| AU (1) | AU2016366846B2 (https=) |
| CA (1) | CA3007476A1 (https=) |
| ES (1) | ES2903000T3 (https=) |
| SG (2) | SG11201804814YA (https=) |
| WO (1) | WO2017100791A1 (https=) |
Families Citing this family (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10834922B2 (en) * | 2014-11-26 | 2020-11-17 | Microban Products Company | Surface disinfectant with residual biocidal property |
| 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 |
| WO2018145111A1 (en) * | 2017-02-06 | 2018-08-09 | Children's Medical Center Corporation | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| US12391936B2 (en) | 2017-03-17 | 2025-08-19 | Rescue Hearing, Inc. | Gene therapy systems and related methods for treatment of hearing loss |
| US20200248203A1 (en) * | 2017-03-17 | 2020-08-06 | Rescue Hearing Inc | Gene Therapy Constructs and Methods for Treatment of Hearing Loss |
| AU2018261769B2 (en) | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| US20210154326A1 (en) * | 2017-06-26 | 2021-05-27 | Arizona Board Of Regents On Behalf Of Arizona State University | CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear |
| EP3661523A4 (en) * | 2017-08-03 | 2021-08-04 | University of Iowa Research Foundation | TREATMENT METHODS FOR GENETIC HEARING LOSS |
| MX2020006435A (es) | 2017-12-19 | 2021-02-09 | Akouos Inc | Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno. |
| DE102018100619A1 (de) | 2018-01-12 | 2019-07-18 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Gentherapeutische Behandlung von Schwerhörigkeit |
| KR20200122320A (ko) | 2018-01-16 | 2020-10-27 | 씨엘에스 테라퓨틱스 리미티드 | 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료 |
| DE102018103924A1 (de) | 2018-02-21 | 2019-08-22 | Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin | Gentherapeutische Behandlung von Schwerhörigkeit |
| JP7430652B2 (ja) * | 2018-03-05 | 2024-02-13 | ザ チルドレンズ メディカル センター コーポレーション | 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 |
| CA3094828A1 (en) | 2018-03-23 | 2019-09-26 | Massachusetts Eye And Ear Infirmary | Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| 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 |
| IL278598B2 (en) | 2018-05-11 | 2025-11-01 | Massachusetts Eye & Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| SG11202012710WA (en) * | 2018-06-25 | 2021-01-28 | Akouos Inc | Methods of treating clrn1-associated hearing loss and/or vision loss |
| WO2020014625A1 (en) * | 2018-07-13 | 2020-01-16 | Akouos, Inc. | Methods of treating non-syndromic sensorineural hearing loss |
| WO2020028717A1 (en) * | 2018-08-01 | 2020-02-06 | University Of Maryland, Baltimore | MODULATION OF mTORC1 ACTIVITY AND AUTOPHAGY VIA CIB2-RHEB INTERACTION |
| WO2020069320A1 (en) | 2018-09-27 | 2020-04-02 | Akouos, Inc. | A method for treating an auditory neuropathy spectrum disorder |
| CA3115810A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
| CA3119059A1 (en) * | 2018-11-07 | 2020-05-14 | Akouos, Inc. | Compositions and methods of inducing differentiation of a hair cell |
| CN118497276A (zh) * | 2018-11-07 | 2024-08-16 | 阿库斯股份有限公司 | 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 |
| WO2020148458A1 (en) * | 2019-01-18 | 2020-07-23 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| 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 |
| US20220202957A1 (en) * | 2019-04-09 | 2022-06-30 | The Regents Of The University Of California | Long-lasting analgesia via targeted in vivo epigenetic repression |
| CN110129368B (zh) * | 2019-04-22 | 2021-03-09 | 中国科学院脑科学与智能技术卓越创新中心 | 一种感染支持细胞和毛细胞的aav载体 |
| US11278013B2 (en) | 2019-04-26 | 2022-03-22 | Usher 2020 Foundation | Large animal model for developing therapeutic agents to treat impaired ophthalmic function in usher syndrome |
| EP3967706A4 (en) * | 2019-05-08 | 2022-12-28 | Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences | AAV MUTANT FOR EFFICIENT INFECTION OF SUPPORT CELLS |
| EP3999647A1 (en) * | 2019-07-18 | 2022-05-25 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| CN110423778B (zh) * | 2019-07-26 | 2023-04-21 | 上海科技大学 | 一种逆行性重组2型腺相关病毒在感染螺旋神经元中的应用 |
| US12570998B2 (en) | 2019-09-09 | 2026-03-10 | Massachusetts Eye & Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV |
| CA3153133A1 (en) * | 2019-09-30 | 2021-04-08 | Chris Bartolome | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| EP4045092A1 (en) * | 2019-10-18 | 2022-08-24 | Research Institute at Nationwide Children's Hospital | Gene therapy targeting cochlear cells |
| CA3159549A1 (en) | 2019-10-30 | 2021-05-06 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2021091950A1 (en) * | 2019-11-04 | 2021-05-14 | Decibel Therapeutics, Inc. | Vestibular supporting cell promoters and uses thereof |
| EP4055175A4 (en) | 2019-11-04 | 2023-11-29 | Decibel Therapeutics, Inc. | Cochlear inner hair cell promoters and uses thereof |
| WO2021108512A1 (en) * | 2019-11-27 | 2021-06-03 | Icahn School Of Medicine At Mount Sinai | Gene therapy for the regeneration of auditory hair cells |
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| 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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| JP2023526047A (ja) * | 2020-05-13 | 2023-06-20 | アコーオス インコーポレイテッド | Ｓｌｃ２６ａ４関連難聴を治療するための組成物及び方法 |
| KR102326582B1 (ko) * | 2020-05-15 | 2021-11-16 | 사회복지법인 삼성생명공익재단 | 청각장애의 진단용 마커 및 그의 용도 |
| WO2022032151A1 (en) * | 2020-08-07 | 2022-02-10 | University Of Miami | Treatment of sensorineural deafness |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
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| JP2025503508A (ja) * | 2021-12-23 | 2025-02-04 | ユニバーシティ オブ ロチェスター | 内耳への薬剤の送達のための組成物及び方法 |
| US20250114480A1 (en) | 2022-01-25 | 2025-04-10 | Bridgebio Gene Therapy Research, Inc. | Gene therapy for genetic hearing loss |
| GB202214972D0 (en) * | 2022-10-11 | 2022-11-23 | Ucl Business Ltd | Products and methods for use in treating NDP-related diseases |
| WO2024151703A2 (en) * | 2023-01-12 | 2024-07-18 | Ohio State Innovation Foundation | Gene therapy for treating hearing loss disorders |
| CN121311586A (zh) * | 2023-05-12 | 2026-01-09 | 苏州星奥拓维生物技术有限公司 | 耳蜗內毛细胞特异性启动子及其应用 |
| AU2024342045A1 (en) | 2023-09-11 | 2026-03-26 | Juntendo Educational Foundation | Aav variant capable of infecting inner ear |
| WO2025085845A1 (en) * | 2023-10-19 | 2025-04-24 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Gene therapy for hearing loss and vestibular disorders |
| US20250276095A1 (en) | 2024-03-04 | 2025-09-04 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
| WO2025226842A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Expression control by drg-expressed mirnas |
| WO2025226841A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Gene therapy approach for treating disorders associated with tnnt2 |
| WO2026011009A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
| WO2026011008A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Expression control by skeletal muscle-expressed mirnas |
| WO2026030242A1 (en) | 2024-07-29 | 2026-02-05 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy |
| WO2026030244A1 (en) | 2024-07-30 | 2026-02-05 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a |
| WO2026050402A1 (en) | 2024-08-29 | 2026-03-05 | Kate Therapeutics, Inc. | Liver de-targeted muscle tropic capsids |
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| US5037384B2 (en) | 1988-01-12 | 1995-12-26 | Cornell Res Foundation Inc | Method and apparatus for the treatment of complicated retinal detachments |
| US20040115621A1 (en) | 2000-02-18 | 2004-06-17 | Allen Rodrigo | Ancestral viruses and vaccines |
| US6692526B1 (en) | 2000-03-15 | 2004-02-17 | Michael E. Snyder | Ophthalmological surgery colorant and delivery system |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| US20050281786A1 (en) | 2004-06-18 | 2005-12-22 | David Poulsen | AAV mediated gene delivery to cochlear cells |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US8388668B2 (en) | 2005-08-05 | 2013-03-05 | Gholam A. Peyman | Methods to regulate polarization of excitable cells |
| WO2011075838A1 (en) | 2009-12-21 | 2011-06-30 | Audigen Inc. | Method of treating or preventing hearing loss |
| WO2013134022A1 (en) | 2012-03-05 | 2013-09-12 | Wake Forest University Health Sciences | Regeneration of inner ear cells |
| HUE052676T2 (hu) * | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| AU2014361781B2 (en) | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| 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 |
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| EP3386537B1 (en) | 2021-10-20 |
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| CN109310745A (zh) | 2019-02-05 |
| US11167042B2 (en) | 2021-11-09 |
| US20180369414A1 (en) | 2018-12-27 |
| KR102794870B1 (ko) | 2025-04-10 |
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