CO2018004564A2 - Genes de ataxia de friedreich modificados y vectores para terapia génica - Google Patents
Genes de ataxia de friedreich modificados y vectores para terapia génicaInfo
- Publication number
- CO2018004564A2 CO2018004564A2 CONC2018/0004564A CO2018004564A CO2018004564A2 CO 2018004564 A2 CO2018004564 A2 CO 2018004564A2 CO 2018004564 A CO2018004564 A CO 2018004564A CO 2018004564 A2 CO2018004564 A2 CO 2018004564A2
- Authority
- CO
- Colombia
- Prior art keywords
- vectors
- gene therapy
- friedreich ataxia
- modified
- genes
- Prior art date
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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
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- 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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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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
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- 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
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- 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
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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
-
- 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)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Physics & Mathematics (AREA)
Abstract
La presente invención se relaciona con genes y vectores modificados de FXN que comprende el gen modificado que proporciona una expresión incrementada de la proteína de frataxina codificada que puede utilizarse para el tratamiento de la ataxia de Friedreich.
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 |
---|---|
CO2018004564A2 true CO2018004564A2 (es) | 2018-12-14 |
Family
ID=57288476
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2018/0004564A CO2018004564A2 (es) | 2015-11-05 | 2018-04-27 | Genes de ataxia de friedreich modificados y vectores para terapia génica |
Country Status (18)
Country | Link |
---|---|
US (2) | US10548947B2 (es) |
EP (1) | EP3370778A1 (es) |
JP (2) | JP6767483B2 (es) |
KR (2) | KR20210005994A (es) |
CN (1) | CN108348621A (es) |
AU (2) | AU2016347887B2 (es) |
BR (1) | BR112018007453A2 (es) |
CA (1) | CA2947584A1 (es) |
CO (1) | CO2018004564A2 (es) |
HK (1) | HK1250924A1 (es) |
IL (1) | IL259141A (es) |
MX (1) | MX2018005084A (es) |
PE (1) | PE20181156A1 (es) |
PH (1) | PH12018500964A1 (es) |
RU (2) | RU2021102893A (es) |
SG (2) | SG10201912763QA (es) |
TW (2) | TWI743442B (es) |
WO (1) | WO2017077451A1 (es) |
Families Citing this family (40)
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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 |
CN108289932A (zh) | 2015-05-22 | 2018-07-17 | D·D·格恩金 | 胞外dna作为神经变性的治疗靶点 |
EP3370778A1 (en) * | 2015-11-05 | 2018-09-12 | Bamboo Therapeutics, Inc. | Modified friedreich ataxia genes and vectors for gene therapy |
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 |
CN109642996A (zh) | 2016-09-21 | 2019-04-16 | 康普技术有限责任公司 | 适配器块组件 |
US11116852B2 (en) | 2016-11-09 | 2021-09-14 | Precigen, Inc. | Frataxin expression constructs |
KR20220010062A (ko) | 2017-10-03 | 2022-01-25 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
MX2020003557A (es) | 2017-10-03 | 2020-10-07 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
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 |
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 |
AU2019209770A1 (en) | 2018-01-16 | 2020-09-03 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
EP3774854A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
JP2021532832A (ja) * | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | フリートライヒ運動失調症を処置するための筋標的化複合体およびそれらの使用 |
CN113166748A (zh) * | 2018-08-10 | 2021-07-23 | 逻辑生物治疗公司 | 用于治疗mma的非破坏性基因疗法 |
SG11202103151RA (en) * | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
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EP3883954A4 (en) * | 2018-11-21 | 2022-08-10 | Stridebio, Inc. | RECOMBINANT VIRAL VECTORS AND NUCLEIC ACIDS FOR THEIR PRODUCTION |
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JP2022520886A (ja) * | 2019-03-27 | 2022-04-01 | シギロン セラピューティクス, インコーポレイテッド | 第vii因子療法のための組成物、デバイス及び方法 |
BR112021019880A2 (pt) | 2019-04-10 | 2022-02-15 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
WO2021003195A1 (en) * | 2019-06-30 | 2021-01-07 | John Fraser Wright | Recombinant aav vectors with altered immunogencity and methods of making the same |
US20220372512A1 (en) * | 2019-07-04 | 2022-11-24 | 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 |
CA3151073A1 (en) * | 2019-09-13 | 2021-03-18 | Darin Falk | Compositions and methods for treatment of friedreich's ataxia |
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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 |
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EP4271420A1 (en) * | 2021-01-04 | 2023-11-08 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
WO2022150772A1 (en) * | 2021-01-11 | 2022-07-14 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
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GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
EP3370778A1 (en) * | 2015-11-05 | 2018-09-12 | Bamboo Therapeutics, Inc. | Modified friedreich ataxia genes and vectors for gene therapy |
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2016
- 2016-11-01 EP EP16795164.9A patent/EP3370778A1/en active Pending
- 2016-11-01 BR BR112018007453A patent/BR112018007453A2/pt not_active IP Right Cessation
- 2016-11-01 AU AU2016347887A patent/AU2016347887B2/en active Active
- 2016-11-01 KR KR1020217000034A patent/KR20210005994A/ko active Application Filing
- 2016-11-01 WO PCT/IB2016/056572 patent/WO2017077451A1/en active Application Filing
- 2016-11-01 SG SG10201912763QA patent/SG10201912763QA/en unknown
- 2016-11-01 US US15/340,721 patent/US10548947B2/en active Active
- 2016-11-01 KR KR1020187015819A patent/KR102200642B1/ko active IP Right Grant
- 2016-11-01 RU RU2021102893A patent/RU2021102893A/ru unknown
- 2016-11-01 RU RU2018116076A patent/RU2743792C2/ru active
- 2016-11-01 CN CN201680064440.0A patent/CN108348621A/zh active Pending
- 2016-11-01 PE PE2018000726A patent/PE20181156A1/es unknown
- 2016-11-01 MX MX2018005084A patent/MX2018005084A/es unknown
- 2016-11-01 JP JP2018521395A patent/JP6767483B2/ja active Active
- 2016-11-01 SG SG11201802408RA patent/SG11201802408RA/en unknown
- 2016-11-04 TW TW108104049A patent/TWI743442B/zh active
- 2016-11-04 TW TW105135911A patent/TWI654301B/zh active
- 2016-11-04 CA CA2947584A patent/CA2947584A1/en active Pending
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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
- 2018-08-13 HK HK18110376.3A patent/HK1250924A1/zh 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 active Active
- 2020-09-17 JP JP2020156320A patent/JP2021019591A/ja active Pending
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