SG11201802408RA - Modified friedreich ataxia genes and vectors for gene therapy - Google Patents
Modified friedreich ataxia genes and vectors for gene therapyInfo
- Publication number
- SG11201802408RA SG11201802408RA SG11201802408RA SG11201802408RA SG11201802408RA SG 11201802408R A SG11201802408R A SG 11201802408RA SG 11201802408R A SG11201802408R A SG 11201802408RA SG 11201802408R A SG11201802408R A SG 11201802408RA SG 11201802408R A SG11201802408R A SG 11201802408RA
- Authority
- SG
- Singapore
- 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
-
- 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 (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 |
|---|---|
| SG11201802408RA true SG11201802408RA (en) | 2018-05-30 |
Family
ID=57288476
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201802408RA SG11201802408RA (en) | 2015-11-05 | 2016-11-01 | Modified friedreich ataxia genes and vectors for gene therapy |
| SG10201912763QA SG10201912763QA (en) | 2015-11-05 | 2016-11-01 | Modified friedreich ataxia genes and vectors for gene therapy |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201912763QA SG10201912763QA (en) | 2015-11-05 | 2016-11-01 | Modified friedreich ataxia genes and vectors for gene therapy |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10548947B2 (OSRAM) |
| EP (1) | EP3370778A1 (OSRAM) |
| JP (2) | JP6767483B2 (OSRAM) |
| KR (2) | KR20210005994A (OSRAM) |
| CN (1) | CN108348621A (OSRAM) |
| AU (2) | AU2016347887B2 (OSRAM) |
| BR (1) | BR112018007453A2 (OSRAM) |
| CA (1) | CA2947584A1 (OSRAM) |
| CO (1) | CO2018004564A2 (OSRAM) |
| HK (1) | HK1250924A1 (OSRAM) |
| IL (1) | IL259141A (OSRAM) |
| MX (1) | MX2018005084A (OSRAM) |
| PE (1) | PE20181156A1 (OSRAM) |
| PH (1) | PH12018500964A1 (OSRAM) |
| RU (2) | RU2021102893A (OSRAM) |
| SG (2) | SG11201802408RA (OSRAM) |
| TW (2) | TWI654301B (OSRAM) |
| WO (1) | WO2017077451A1 (OSRAM) |
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 |
|---|---|---|
| IL259141A (en) | Modified friedreich ataxia genes and vectors for gene therapy | |
| IL304084B1 (en) | Gene healing | |
| IL260796B1 (en) | Factor VIII genes are effective | |
| ZA201606763B (en) | Aav vectors for retinal and cns gene therapy | |
| PT3612624T (pt) | Terapia génica | |
| IL258005A (en) | Methods and materials for gene therapy for beta-4,1-en.-acetylgalactosaminyltransferase 2 | |
| IL263009A (en) | Optimal clni genes and expression cassettes and their use | |
| LT3612237T (lt) | Genų terapija | |
| IL257831B2 (en) | Diplosporic garden | |
| GB201407322D0 (en) | Gene therapy | |
| GB201707212D0 (en) | Gene therapy for ciliopathies | |
| IL257500A (en) | Altered cullin1 gene | |
| PL3638796T3 (pl) | Wektory ekspresyjne zawierające zmodyfikowane geny | |
| GB2572716B (en) | Gene Therapy Vector System and Prodrug Genes | |
| GB201701968D0 (en) | Gene therapy | |
| GB201513151D0 (en) | Functional gene replacement therapy | |
| GB201704634D0 (en) | Gene therapy | |
| GB201616821D0 (en) | Gene therapy | |
| GB201612105D0 (en) | Gene therapy | |
| GB201612104D0 (en) | Gene therapy | |
| SG10201912076UA (en) | Gene therapy composition | |
| GB201417148D0 (en) | Eukaryotic expression vector encoding canine interleukin 12 for cancer gene therapy and the use thereof |