IL259141A - Modified friedreich ataxia genes and vectors for gene therapy - Google Patents
Modified friedreich ataxia genes and vectors for gene therapyInfo
- Publication number
- IL259141A IL259141A IL259141A IL25914118A IL259141A IL 259141 A IL259141 A IL 259141A IL 259141 A IL259141 A IL 259141A IL 25914118 A IL25914118 A IL 25914118A IL 259141 A IL259141 A IL 259141A
- Authority
- IL
- Israel
- Prior art keywords
- vectors
- gene therapy
- friedreich ataxia
- modified
- ataxia genes
- Prior art date
Links
- 208000024412 Friedreich ataxia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
Classifications
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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
-
- 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
- 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
-
- 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)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Physics & Mathematics (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562251288P | 2015-11-05 | 2015-11-05 | |
| US201662411980P | 2016-10-24 | 2016-10-24 | |
| PCT/IB2016/056572 WO2017077451A1 (en) | 2015-11-05 | 2016-11-01 | Modified friedreich ataxia genes and vectors for gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL259141A true IL259141A (en) | 2018-06-28 |
Family
ID=57288476
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL259141A IL259141A (en) | 2015-11-05 | 2018-05-03 | Modified friedreich ataxia genes and vectors for gene therapy |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (enExample) |
| EP (1) | EP3370778A1 (enExample) |
| JP (2) | JP6767483B2 (enExample) |
| KR (2) | KR102200642B1 (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) | SG10201912763QA (enExample) |
| TW (2) | TWI743442B (enExample) |
| WO (1) | WO2017077451A1 (enExample) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3286321A4 (en) * | 2015-04-24 | 2018-10-17 | 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 |
| RU2726131C2 (ru) | 2015-05-22 | 2020-07-09 | Дмитрий Дмитриевич Генкин | Внеклеточная днк в качестве терапевтической мишени при нейродегенерации |
| SG10201912763QA (en) * | 2015-11-05 | 2020-02-27 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| AU2017237912B2 (en) * | 2016-03-23 | 2022-03-10 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| EP3491440A4 (en) | 2016-09-21 | 2020-02-26 | Commscope Technologies LLC | ADAPTER BLOCK ASSEMBLY |
| AU2017359342B2 (en) | 2016-11-09 | 2022-02-17 | Intrexon Corporation | Frataxin expression constructs |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| CN111542549A (zh) * | 2017-10-03 | 2020-08-14 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| EP3692158A4 (en) | 2017-10-03 | 2021-08-04 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| MX2020004005A (es) | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| PL3697914T3 (pl) * | 2017-10-17 | 2023-10-16 | Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol | Wektory do leczenia ataksji friedreicha |
| EP3740581A4 (en) | 2018-01-16 | 2021-10-27 | CLS Therapeutics Limited | TREATMENT OF DISEASES BY LIVER EXPRESSION OF AN ENZYME WITH DESOXYRIBONUCLEASE (DNASE) ACTIVITY |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| CA3108386A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating friedreich's ataxia |
| 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 |
| WO2020032986A1 (en) * | 2018-08-10 | 2020-02-13 | Logicbio Therapeutics, Inc. | Non-disruptive gene therapy for the treatment of mma |
| US12281321B2 (en) * | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3880179A2 (en) * | 2018-11-14 | 2021-09-22 | 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 |
| MX2021005997A (es) * | 2018-11-21 | 2021-08-11 | Vectores virales recombinantes y acidos nucleicos para producirlos. | |
| AU2019406214A1 (en) * | 2018-12-21 | 2021-08-05 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| CA3133357A1 (en) * | 2019-03-27 | 2020-10-01 | Sigilon Therapeutics, Inc. | Compositions, devices and methods for factor vii therapy |
| MX2021011757A (es) * | 2019-03-28 | 2021-12-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la expresión de polipéptidos. |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| AU2020300519A1 (en) * | 2019-06-30 | 2022-02-24 | 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 |
| 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 |
| 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 |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| BR112022011916A2 (pt) * | 2019-12-19 | 2022-09-06 | Univ Pennsylvania | Composições para tratar a ataxia de friedreich |
| 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| 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 |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス， インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| AU2021375404A1 (en) | 2020-11-03 | 2023-06-08 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| 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 |
| 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 |
| EP4551596A4 (en) * | 2022-07-06 | 2026-07-22 | Adverum Biotechnologies Inc | COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY |
| CN116121350A (zh) * | 2022-12-02 | 2023-05-16 | 百码科技(深圳)有限公司 | 一种检测fxn致病基因突变的方法 |
| 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 |
| WO2026017965A1 (en) | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
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 |
| JP4108746B2 (ja) | 1995-06-07 | 2008-06-25 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ヘルパーウイルスを含まないａａｖ産生 |
| EP1696036B1 (en) | 1996-09-06 | 2010-04-21 | The Trustees of The University of Pennsylvania | Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | 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 |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| DE69936104T2 (de) | 1998-11-05 | 2008-01-17 | The Trustees Of The University Of Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| CA2399576A1 (en) | 2000-12-07 | 2002-06-13 | Universite De Nantes | Inducible highly productive raav packaging cell-lines |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| CN101848936B (zh) * | 2007-08-20 | 2017-05-03 | 葛兰素集团有限公司 | 生产方法 |
| 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 |
| CA2784861A1 (en) * | 2009-12-21 | 2011-07-14 | Pharmathene, Inc. | Recombinant butyrylcholinesterases and truncates thereof |
| WO2011097640A1 (en) * | 2010-02-08 | 2011-08-11 | 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 |
| WO2012170771A1 (en) * | 2011-06-09 | 2012-12-13 | Curna, Inc. | Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn |
| EP2771455B1 (en) | 2011-10-28 | 2016-10-05 | 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 |
| JP2016517278A (ja) | 2013-03-15 | 2016-06-16 | 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 | スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法 |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| WO2015006743A1 (en) | 2013-07-12 | 2015-01-15 | The Children's Hospital Of Philadelphia | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
| NZ716102A (en) | 2013-07-22 | 2021-12-24 | The Children’S Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| SG10201912763QA (en) * | 2015-11-05 | 2020-02-27 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
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2016
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en not_active Ceased
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja not_active Expired - Fee Related
- 2016-11-01 HK HK18110376.3A patent/HK1250924A1/zh unknown
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko not_active Expired - Fee Related
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en not_active Withdrawn
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en not_active Ceased
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko not_active Withdrawn
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh not_active IP Right Cessation
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh not_active IP Right Cessation
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2018
- 2018-04-27 CO CONC2018/0004564A patent/CO2018004564A2/es unknown
- 2018-05-03 IL IL259141A patent/IL259141A/en unknown
- 2018-05-03 PH PH12018500964A patent/PH12018500964A1/en unknown
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2019
- 2019-12-16 US US16/716,033 patent/US20200384073A1/en not_active Abandoned
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2020
- 2020-02-11 AU AU2020200976A patent/AU2020200976B2/en not_active Ceased
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
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