RU2021102893A - Модифицированные гены атаксии фридрейха и векторы для генной терапии - Google Patents
Модифицированные гены атаксии фридрейха и векторы для генной терапии Download PDFInfo
- Publication number
- 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
- Authority
- RU
- Russia
- Prior art keywords
- nucleic acid
- seq
- acid sequence
- sequence
- aav2
- Prior art date
Links
- 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
-
- 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
-
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/28—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
-
- 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
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- 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)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- 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)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (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)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (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 (enExample) |
| EP (1) | EP3370778A1 (enExample) |
| JP (2) | JP6767483B2 (enExample) |
| KR (2) | KR20210005994A (enExample) |
| CN (1) | CN108348621A (enExample) |
| AU (2) | AU2016347887B2 (enExample) |
| BR (1) | BR112018007453A2 (enExample) |
| CA (1) | CA2947584A1 (enExample) |
| CO (1) | CO2018004564A2 (enExample) |
| HK (1) | HK1250924A1 (enExample) |
| IL (1) | IL259141A (enExample) |
| MX (1) | MX2018005084A (enExample) |
| PE (1) | PE20181156A1 (enExample) |
| PH (1) | PH12018500964A1 (enExample) |
| RU (2) | RU2021102893A (enExample) |
| SG (2) | SG11201802408RA (enExample) |
| TW (2) | TWI654301B (enExample) |
| WO (1) | WO2017077451A1 (enExample) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10617770B2 (en) * | 2015-04-24 | 2020-04-14 | University Of Florida Research Foundation, Incorporated | AAV vector for treatment of Friedreich's ataxia |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| ES2799515T3 (es) | 2015-05-22 | 2020-12-18 | Dmitry Dmitrievich Genkin | ADN extracelular como una diana terapéutica en la neurodegeneración |
| KR20210005994A (ko) * | 2015-11-05 | 2021-01-15 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| ES3038656T3 (en) * | 2016-03-23 | 2025-10-14 | Univ California | Methods of treating mitochondrial disorders |
| CN109642996A (zh) | 2016-09-21 | 2019-04-16 | 康普技术有限责任公司 | 适配器块组件 |
| AU2017359342B2 (en) * | 2016-11-09 | 2022-02-17 | Intrexon Corporation | Frataxin expression constructs |
| CA3075656A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| KR20200078513A (ko) | 2017-10-03 | 2020-07-01 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| JP7361037B2 (ja) | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| US11802294B2 (en) * | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7128491B2 (ja) * | 2017-10-17 | 2022-08-31 | フンダシオ インスティテュート ディンベスティガシオ エン シエンシエス デ ラ サリュ ジャーマンス トライアス アイ プジョル | フリードライヒ運動失調症の治療用のベクター |
| JP2021510539A (ja) | 2018-01-16 | 2021-04-30 | シーエルエス セラピューティクス リミテッドＣＬＳ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ | デオキシリボヌクレアーゼ（ＤＮａｓｅ）活性を有する酵素の肝臓発現による疾患の処置 |
| SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| AU2019247191A1 (en) | 2018-04-03 | 2020-10-15 | Ginkgo Bioworks, Inc. | Virus vectors for targeting ophthalmic tissues |
| JP2021519581A (ja) | 2018-04-03 | 2021-08-12 | ストライドバイオ，インコーポレイテッド | 抗体を回避するウイルスベクター |
| 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 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| WO2020028840A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating friedreich's ataxia |
| WO2020032986A1 (en) * | 2018-08-10 | 2020-02-13 | Logicbio Therapeutics, Inc. | Non-disruptive gene therapy for the treatment of mma |
| AU2019346655B2 (en) * | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US20220008557A1 (en) * | 2018-11-14 | 2022-01-13 | Flagship Pioneering Innovations V, Inc. | Fusosome compositions for cns delivery |
| EP3880823A4 (en) * | 2018-11-16 | 2022-08-17 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| TW202039533A (zh) * | 2018-11-21 | 2020-11-01 | 美商史崔德生物公司 | 重組病毒載體及用於生產其之核酸 |
| EP3897689B1 (en) * | 2018-12-21 | 2025-02-05 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| US20230123802A1 (en) * | 2019-03-27 | 2023-04-20 | Sigilon Therapeutics, Inc. | Compositions, devices, and methods for factor vii therapy |
| TW202102529A (zh) * | 2019-03-28 | 2021-01-16 | 美商英特利亞醫療公司 | 用於多肽表現之多核苷酸、組合物及方法 |
| BR112021019880A2 (pt) | 2019-04-10 | 2022-02-15 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
| AU2020300519A1 (en) * | 2019-06-30 | 2022-02-24 | John Fraser Wright | Recombinant AAV vectors with altered immunogencity and methods of making the same |
| EP3999120A4 (en) * | 2019-07-04 | 2024-02-21 | Children's Medical Research Institute | Methods and aav vectors for in vivo transduction |
| WO2021011368A1 (en) * | 2019-07-12 | 2021-01-21 | Dmitry Dmitrievich Genkin | Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity |
| EP4028027A4 (en) * | 2019-09-13 | 2023-11-01 | Lacerta Therapeutics, Inc | COMPOSITIONS AND METHODS FOR TREATING FRIEDREICH AAXIA |
| CA3157700A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| IL293972A (en) * | 2019-12-19 | 2022-08-01 | Univ Pennsylvania | Compositions for treating friedreich's ataxia |
| 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 |
| WO2022026516A1 (en) * | 2020-07-29 | 2022-02-03 | 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 |
| CA3189878A1 (en) | 2020-08-19 | 2022-02-24 | Colin O'BANION | Adeno-associated virus vectors for treatment of rett syndrome |
| MX2023005218A (es) | 2020-11-03 | 2023-05-16 | Pfizer | Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico. |
| 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 |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024011109A2 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies, Inc. | Compositions and methods for treatment of achromotopsia |
| CN117089514A (zh) * | 2023-10-13 | 2023-11-21 | 思鹏生物科技(苏州)有限公司 | 提升hek293细胞系aav生产效率的细胞筛选驯化方法 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| DE69627532T2 (de) | 1995-06-07 | 2004-01-29 | Univ North Carolina | Helfervirus-freie herstellung von aav |
| CA2264483C (en) | 1996-09-06 | 2011-03-22 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| AU768729B2 (en) | 1998-11-05 | 2004-01-08 | Trustees Of The University Of Pennsylvania, The | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| EP1339861A2 (en) | 2000-12-07 | 2003-09-03 | Universite de Nantes | Inducible highly productive raav packaging cell-lines |
| ES2874298T3 (es) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| DK3216802T3 (da) * | 2007-08-20 | 2021-01-04 | Glaxo Group Ltd | Produktionsfremgangsmåde |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US8729245B2 (en) * | 2009-12-21 | 2014-05-20 | Pharmathene, Inc. | Recombinant butyrylcholinesterases and truncates thereof |
| CA2788902C (en) * | 2010-02-08 | 2019-09-03 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecules encoding rantes, and compositions comprising and methods of using the same |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| US9593330B2 (en) * | 2011-06-09 | 2017-03-14 | Curna, Inc. | Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN |
| US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| US9066966B2 (en) | 2013-02-01 | 2015-06-30 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia |
| WO2014144486A2 (en) | 2013-03-15 | 2014-09-18 | The Children's Hospital Of Philadelphia | Vectors comprising stuffer/filler polynucleotide sequences and methods of use |
| CN110885854A (zh) | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| CN105247044B (zh) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | 腺相关病毒变体及其使用方法 |
| KR102445330B1 (ko) | 2013-07-12 | 2022-09-19 | 더 칠드런스 호스피탈 오브 필라델피아 | Ａａｖ 벡터 및 항―ａａｖ （아데노-관련 바이러스） 중화 항체에 대한 검정 |
| MX2016001044A (es) | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| WO2015054653A2 (en) | 2013-10-11 | 2015-04-16 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| KR20210005994A (ko) * | 2015-11-05 | 2021-01-15 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
-
2016
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko not_active Withdrawn
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en not_active Ceased
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en not_active Withdrawn
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja not_active Expired - Fee Related
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 HK HK18110376.3A patent/HK1250924A1/zh unknown
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko not_active Expired - Fee Related
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en not_active Ceased
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh not_active IP Right Cessation
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh not_active IP Right Cessation
-
2018
- 2018-04-27 CO CONC2018/0004564A patent/CO2018004564A2/es unknown
- 2018-05-03 PH PH12018500964A patent/PH12018500964A1/en unknown
- 2018-05-03 IL IL259141A patent/IL259141A/en unknown
-
2019
- 2019-12-16 US US16/716,033 patent/US20200384073A1/en not_active Abandoned
-
2020
- 2020-02-11 AU AU2020200976A patent/AU2020200976B2/en not_active Ceased
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2021102893A (ru) | Модифицированные гены атаксии фридрейха и векторы для генной терапии | |
| JP2021019591A5 (enExample) | ||
| JP2018531609A5 (enExample) | ||
| ES2768763T3 (es) | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR | |
| JP6619454B2 (ja) | キャプシド | |
| JP7162021B2 (ja) | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 | |
| US10413598B2 (en) | Factor IX gene therapy | |
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| US20040052764A1 (en) | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids | |
| JP2020522269A5 (enExample) | ||
| JP2020528734A5 (enExample) | ||
| JP2020537542A5 (enExample) | ||
| RU2020109343A (ru) | Полинуклеотиды aadc для лечения болезни паркинсона | |
| RU2017125234A (ru) | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний | |
| JPWO2020106916A5 (enExample) | ||
| WO2020208032A1 (en) | Hybrid promoters for muscle expression | |
| US20240067678A1 (en) | Adeno-associated virus capsids and vectors | |
| US20220042045A1 (en) | Expression cassettes for gene therapy vectors | |
| JP2024543253A (ja) | Ｓｌｃ６ａ１遺伝子治療のための材料及び方法 | |
| WO2023012313A1 (en) | Hybrid promoters for gene expression in muscles and in the cns | |
| CN118064436B (zh) | 一种增强基因表达的嵌合启动子 | |
| JPWO2021221995A5 (enExample) | ||
| JP2023059858A5 (enExample) | ||
| RU2825667C2 (ru) | Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе | |
| CN116179605B (zh) | 一种重组腺相关病毒载体及其应用 |