RU2021102893A - Модифицированные гены атаксии фридрейха и векторы для генной терапии - Google Patents
Модифицированные гены атаксии фридрейха и векторы для генной терапии Download PDFInfo
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- RU2021102893A RU2021102893A RU2021102893A RU2021102893A RU2021102893A RU 2021102893 A RU2021102893 A RU 2021102893A RU 2021102893 A RU2021102893 A RU 2021102893A RU 2021102893 A RU2021102893 A RU 2021102893A RU 2021102893 A RU2021102893 A RU 2021102893A
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- 239000013598 vector Substances 0.000 title claims 8
- 108090000623 proteins and genes Proteins 0.000 title claims 7
- 206010003591 Ataxia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 claims 61
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 31
- 108020004707 nucleic acids Proteins 0.000 claims 30
- 102000039446 nucleic acids Human genes 0.000 claims 30
- 239000013608 rAAV vector Substances 0.000 claims 21
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 17
- 102000003869 Frataxin Human genes 0.000 claims 17
- 108090000217 Frataxin Proteins 0.000 claims 17
- 210000000234 capsid Anatomy 0.000 claims 12
- 208000024412 Friedreich ataxia Diseases 0.000 claims 10
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 10
- 238000000034 method Methods 0.000 claims 10
- 102000004169 proteins and genes Human genes 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 150000001413 amino acids Chemical group 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 108020004705 Codon Proteins 0.000 claims 1
- 102000008186 Collagen Human genes 0.000 claims 1
- 108010035532 Collagen Proteins 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 108700042658 GAP-43 Proteins 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 101710150114 Protein rep Proteins 0.000 claims 1
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims 1
- 101710152114 Replication protein Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 238000004113 cell culture Methods 0.000 claims 1
- 229920001436 collagen Polymers 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 238000007917 intracranial administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000006641 stabilisation Effects 0.000 claims 1
- 238000011105 stabilization Methods 0.000 claims 1
- 238000004114 suspension culture Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 230000002463 transducing effect Effects 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Classifications
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- 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
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- 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
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- 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
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- 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
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- A61P25/00—Drugs for disorders of the nervous system
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- 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
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- 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
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- General Engineering & Computer Science (AREA)
- Marine Sciences & Fisheries (AREA)
- Biophysics (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Plant Pathology (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251288P | 2015-11-05 | 2015-11-05 | |
| US62/251.288 | 2015-11-05 | ||
| US201662411980P | 2016-10-24 | 2016-10-24 | |
| US62/411.980 | 2016-10-24 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018116076A Division RU2743792C2 (ru) | 2015-11-05 | 2016-11-01 | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2021102893A true RU2021102893A (ru) | 2021-03-03 |
Family
ID=57288476
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2021102893A RU2021102893A (ru) | 2015-11-05 | 2016-11-01 | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| RU2018116076A RU2743792C2 (ru) | 2015-11-05 | 2016-11-01 | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2018116076A RU2743792C2 (ru) | 2015-11-05 | 2016-11-01 | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (https=) |
| EP (1) | EP3370778A1 (https=) |
| JP (2) | JP6767483B2 (https=) |
| KR (2) | KR20210005994A (https=) |
| CN (1) | CN108348621A (https=) |
| AU (2) | AU2016347887B2 (https=) |
| BR (1) | BR112018007453A2 (https=) |
| CA (1) | CA2947584A1 (https=) |
| CO (1) | CO2018004564A2 (https=) |
| HK (1) | HK1250924A1 (https=) |
| IL (1) | IL259141A (https=) |
| MX (1) | MX2018005084A (https=) |
| PE (1) | PE20181156A1 (https=) |
| PH (1) | PH12018500964A1 (https=) |
| RU (2) | RU2021102893A (https=) |
| SG (2) | SG10201912763QA (https=) |
| TW (2) | TWI743442B (https=) |
| WO (1) | WO2017077451A1 (https=) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016172659A1 (en) * | 2015-04-24 | 2016-10-27 | University Of Florida Research Foundation, Inc. | Aav vector for treatment of friedreich's ataxia |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2016190780A1 (en) | 2015-05-22 | 2016-12-01 | Dmitry Dmitrievich Genkin | Extracellular dna as a therapeutic target in neurodegeneration |
| RU2021102893A (ru) * | 2015-11-05 | 2021-03-03 | Бамбу Терапьютикс, Инк. | Модифицированные гены атаксии фридрейха и векторы для генной терапии |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| CN116059245A (zh) * | 2016-03-23 | 2023-05-05 | 加利福尼亚大学董事会 | 治疗线粒体障碍的方法 |
| US10935747B2 (en) | 2016-09-21 | 2021-03-02 | Commscope Technologies Llc | Adapter block assembly |
| CN110139933B (zh) * | 2016-11-09 | 2024-03-08 | 英特瑞克斯顿股份有限公司 | 共济蛋白表达构建体 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| WO2019076973A1 (en) * | 2017-10-17 | 2019-04-25 | Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol | VECTORS FOR THE TREATMENT OF FRIEDREICH ATAXIA |
| KR20200122320A (ko) | 2018-01-16 | 2020-10-27 | 씨엘에스 테라퓨틱스 리미티드 | 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| KR20210086601A (ko) * | 2018-08-02 | 2021-07-08 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 프리드라이히 운동실조를 치료하기 위한 그의 용도 |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| KR20210049833A (ko) * | 2018-08-10 | 2021-05-06 | 로직바이오 테라퓨틱스, 인크. | Mma의 치료를 위한 비-붕괴적 유전자 요법 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| SG11202105079QA (en) * | 2018-11-14 | 2021-06-29 | Flagship Pioneering Innovations V Inc | Fusosome compositions for cns delivery |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| AU2019385506A1 (en) * | 2018-11-21 | 2021-06-03 | Takeda Pharmaceutical Company Limited | Recombinant viral vectors and nucleic acids for producing the same |
| CA3124415A1 (en) * | 2018-12-21 | 2020-06-25 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| CN114222590A (zh) * | 2019-03-27 | 2022-03-22 | 西吉隆医疗股份有限公司 | 用于因子vii疗法的组合物、装置和方法 |
| BR112021019224A2 (pt) * | 2019-03-28 | 2021-11-30 | Intellia Therapeutics Inc | Polinucleotídeos, composições e métodos para expressão de polipeptídeo |
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| WO2021003195A1 (en) * | 2019-06-30 | 2021-01-07 | John Fraser Wright | Recombinant aav vectors with altered immunogencity and methods of making the same |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| CA3147034A1 (en) * | 2019-07-12 | 2021-01-21 | Cls Therapeutics Limited | Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity |
| JP2022548270A (ja) * | 2019-09-13 | 2022-11-17 | ラセルタ セラピューティクス， インコーポレイテッド | フリードライヒ運動失調症の治療のための組成物及び方法 |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4085144A4 (en) * | 2019-12-19 | 2024-03-27 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH AAXIA |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| US11946065B2 (en) | 2020-07-29 | 2024-04-02 | The Board Of Regents Of The University Of Texas System | Transgene cassettes, AAV vectors, and AAV viral vectors for expression of human codon-optimized CSTB |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| JP2023548816A (ja) | 2020-11-03 | 2023-11-21 | ファイザー・インク | 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法 |
| JP2024503234A (ja) | 2020-12-23 | 2024-01-25 | ファイザー・インク | 親和性クロマトグラフィーによるａａｖベクターの精製方法 |
| US20240058477A1 (en) * | 2021-01-04 | 2024-02-22 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| EP4274592A4 (en) * | 2021-01-11 | 2025-03-19 | The Trustees of The University of Pennsylvania | COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
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