MX2020010477A - Composiciones y metodos para el tratamiento de la distrofia macular. - Google Patents
Composiciones y metodos para el tratamiento de la distrofia macular.Info
- Publication number
- MX2020010477A MX2020010477A MX2020010477A MX2020010477A MX2020010477A MX 2020010477 A MX2020010477 A MX 2020010477A MX 2020010477 A MX2020010477 A MX 2020010477A MX 2020010477 A MX2020010477 A MX 2020010477A MX 2020010477 A MX2020010477 A MX 2020010477A
- Authority
- MX
- Mexico
- Prior art keywords
- macular dystrophy
- compositions
- methods
- treating macular
- sequence encoding
- Prior art date
Links
Classifications
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K48/0083—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 administration regime
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Abstract
La descripción proporciona una composición que comprende una secuencia de ácido nucleico que comprende (a) una secuencia que codifica un promotor de distrofia macular viteliforme 2 (VMD2), y (b) una secuencia que codifica una proteína bestrofina-1 (BEST1), así como el uso de estas composiciones para el tratamiento de la distrofia macular en un sujeto, que comprende la administración de la composición al ojo de un sujeto por una via subretiniana o supracoroidea.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862653131P | 2018-04-05 | 2018-04-05 | |
PCT/US2019/026062 WO2019195727A1 (en) | 2018-04-05 | 2019-04-05 | Compositions and methods for treating macular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020010477A true MX2020010477A (es) | 2021-03-02 |
Family
ID=66223872
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020010477A MX2020010477A (es) | 2018-04-05 | 2019-04-05 | Composiciones y metodos para el tratamiento de la distrofia macular. |
Country Status (21)
Country | Link |
---|---|
US (2) | US20190307900A1 (es) |
EP (1) | EP3775233A1 (es) |
JP (1) | JP2021520232A (es) |
KR (1) | KR20210005040A (es) |
CN (1) | CN113056561A (es) |
AU (1) | AU2019247864A1 (es) |
BR (1) | BR112020020204A2 (es) |
CA (1) | CA3096088A1 (es) |
CL (1) | CL2020002561A1 (es) |
CO (1) | CO2020013690A2 (es) |
EA (1) | EA202092069A1 (es) |
IL (1) | IL277779A (es) |
JO (1) | JOP20200253A1 (es) |
MA (1) | MA52199A (es) |
MX (1) | MX2020010477A (es) |
PE (1) | PE20210918A1 (es) |
PH (1) | PH12020551641A1 (es) |
RU (1) | RU2020132890A (es) |
SG (1) | SG11202009759SA (es) |
TW (1) | TW202003052A (es) |
WO (1) | WO2019195727A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20210086645A (ko) * | 2018-10-25 | 2021-07-08 | 박스알타 인코퍼레이티드 | Aav 삼중-플라스미드 시스템 |
WO2020140007A1 (en) * | 2018-12-28 | 2020-07-02 | University Of Rochester | Gene therapy for best1 dominant mutations |
EP4110466A4 (en) * | 2020-02-28 | 2024-03-27 | Univ Pennsylvania | TREATMENT OF AUTOSOMAL DOMINANT BESTROPHINOPATHIES AND METHODS OF EVALUATION THEREOF |
CN111849998A (zh) * | 2020-07-29 | 2020-10-30 | 武汉纽福斯生物科技有限公司 | 编码人卵黄状黄斑病蛋白1的核酸分子及其应用 |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN103993040B (zh) * | 2008-06-18 | 2018-02-13 | 牛津生物医学(英国)有限公司 | 病毒纯化 |
US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
CN105960413B (zh) * | 2013-11-20 | 2020-03-27 | 泰莱托恩基金会 | 人工dna-结合蛋白及其用途 |
KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
SI3265571T1 (sl) * | 2015-03-03 | 2022-10-28 | Fondazione Telethon | Več vektorski sistem in njegove uporabe |
EP3374494A4 (en) * | 2015-11-11 | 2019-05-01 | Coda Biotherapeutics, Inc. | CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY |
-
2019
- 2019-04-05 RU RU2020132890A patent/RU2020132890A/ru unknown
- 2019-04-05 CN CN201980031769.0A patent/CN113056561A/zh not_active Withdrawn
- 2019-04-05 WO PCT/US2019/026062 patent/WO2019195727A1/en active Application Filing
- 2019-04-05 JO JOP/2020/0253A patent/JOP20200253A1/ar unknown
- 2019-04-05 CA CA3096088A patent/CA3096088A1/en not_active Withdrawn
- 2019-04-05 MA MA052199A patent/MA52199A/fr unknown
- 2019-04-05 EA EA202092069A patent/EA202092069A1/ru unknown
- 2019-04-05 BR BR112020020204-5A patent/BR112020020204A2/pt not_active Application Discontinuation
- 2019-04-05 JP JP2021504131A patent/JP2021520232A/ja not_active Withdrawn
- 2019-04-05 US US16/376,808 patent/US20190307900A1/en not_active Abandoned
- 2019-04-05 MX MX2020010477A patent/MX2020010477A/es unknown
- 2019-04-05 KR KR1020207031540A patent/KR20210005040A/ko unknown
- 2019-04-05 PE PE2020001530A patent/PE20210918A1/es unknown
- 2019-04-05 AU AU2019247864A patent/AU2019247864A1/en not_active Abandoned
- 2019-04-05 EP EP19718557.2A patent/EP3775233A1/en not_active Withdrawn
- 2019-04-05 SG SG11202009759SA patent/SG11202009759SA/en unknown
- 2019-04-08 TW TW108112207A patent/TW202003052A/zh unknown
-
2020
- 2020-10-02 CL CL2020002561A patent/CL2020002561A1/es unknown
- 2020-10-04 IL IL277779A patent/IL277779A/en unknown
- 2020-10-05 PH PH12020551641A patent/PH12020551641A1/en unknown
- 2020-10-30 CO CONC2020/0013690A patent/CO2020013690A2/es unknown
-
2022
- 2022-09-15 US US17/945,344 patent/US20230149566A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
EA202092069A1 (ru) | 2021-03-12 |
MA52199A (fr) | 2021-02-17 |
CO2020013690A2 (es) | 2021-04-19 |
SG11202009759SA (en) | 2020-10-29 |
EP3775233A1 (en) | 2021-02-17 |
CL2020002561A1 (es) | 2021-04-23 |
RU2020132890A (ru) | 2022-05-06 |
JP2021520232A (ja) | 2021-08-19 |
US20190307900A1 (en) | 2019-10-10 |
JOP20200253A1 (ar) | 2020-10-04 |
PH12020551641A1 (en) | 2021-07-26 |
AU2019247864A1 (en) | 2020-10-22 |
WO2019195727A1 (en) | 2019-10-10 |
KR20210005040A (ko) | 2021-01-13 |
US20230149566A1 (en) | 2023-05-18 |
CN113056561A (zh) | 2021-06-29 |
BR112020020204A2 (pt) | 2021-01-19 |
IL277779A (en) | 2020-11-30 |
TW202003052A (zh) | 2020-01-16 |
PE20210918A1 (es) | 2021-05-19 |
CA3096088A1 (en) | 2019-10-10 |
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