PE20210918A1 - Composiciones y metodos para el tratamiento de la distrofia macular - Google Patents
Composiciones y metodos para el tratamiento de la distrofia macularInfo
- Publication number
- PE20210918A1 PE20210918A1 PE2020001530A PE2020001530A PE20210918A1 PE 20210918 A1 PE20210918 A1 PE 20210918A1 PE 2020001530 A PE2020001530 A PE 2020001530A PE 2020001530 A PE2020001530 A PE 2020001530A PE 20210918 A1 PE20210918 A1 PE 20210918A1
- Authority
- PE
- Peru
- Prior art keywords
- macular dystrophy
- composition
- treatment
- compositions
- methods
- 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
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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
- A61K48/0083—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
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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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- 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
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- 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
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- 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
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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
- 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 divulgacion proporciona una composicion que comprende una secuencia de acido nucleico que comprende (a) una secuencia que codifica un promotor de distrofia macular viteliforme 2 (VMD2) de preferencia humano que comprende la secuencia de nucleotidos SEQ ID NO° 1, y (b) una secuencia que codifica una proteina bestrofina-1 (BEST1) de preferencia humano que comprende la secuencia de nucleotidos SEQ ID NO° 3. Tambien refiere a un vector, una celula y composicion farmaceutica que comprenden la citada composicion; siendo util para el tratamiento de la distrofia macular en un sujeto mediante la administracion de la composicion 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 |
---|---|
PE20210918A1 true PE20210918A1 (es) | 2021-05-19 |
Family
ID=66223872
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2020001530A PE20210918A1 (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 |
---|---|---|---|---|
AU2019363593A1 (en) * | 2018-10-25 | 2021-04-29 | Takeda Pharmaceutical Company Limited | AAV triple-plasmid system |
WO2020140007A1 (en) * | 2018-12-28 | 2020-07-02 | University Of Rochester | Gene therapy for best1 dominant mutations |
US20230112568A1 (en) * | 2020-02-28 | 2023-04-13 | The Trustees Of The University Of Pennsylvania | Treating autosomal recessive bestrophinopathies and methods for evaluating same |
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 |
---|---|---|---|---|
ES2762864T3 (es) * | 2008-06-18 | 2020-05-26 | Oxford Biomedica Ltd | Purificación de virus |
US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
CA2930282A1 (en) * | 2013-11-20 | 2015-05-28 | Fondazione Telethon | Artificial dna-binding proteins and uses thereof |
EP3077520A1 (en) * | 2013-12-06 | 2016-10-12 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject |
GB201322798D0 (en) * | 2013-12-20 | 2014-02-05 | Oxford Biomedica Ltd | Production system |
AU2016227668B2 (en) * | 2015-03-03 | 2019-06-27 | Fondazione Telethon | Multiple vector system and uses thereof |
EP3374494A4 (en) * | 2015-11-11 | 2019-05-01 | Coda Biotherapeutics, Inc. | CRISPR COMPOSITIONS AND METHODS OF USE FOR GENE THERAPY |
-
2019
- 2019-04-05 AU AU2019247864A patent/AU2019247864A1/en not_active Abandoned
- 2019-04-05 EA EA202092069A patent/EA202092069A1/ru unknown
- 2019-04-05 WO PCT/US2019/026062 patent/WO2019195727A1/en active Application Filing
- 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 RU RU2020132890A patent/RU2020132890A/ru unknown
- 2019-04-05 KR KR1020207031540A patent/KR20210005040A/ko unknown
- 2019-04-05 MA MA052199A patent/MA52199A/fr unknown
- 2019-04-05 PE PE2020001530A patent/PE20210918A1/es unknown
- 2019-04-05 JO JOP/2020/0253A patent/JOP20200253A1/ar unknown
- 2019-04-05 BR BR112020020204-5A patent/BR112020020204A2/pt not_active Application Discontinuation
- 2019-04-05 CN CN201980031769.0A patent/CN113056561A/zh not_active Withdrawn
- 2019-04-05 EP EP19718557.2A patent/EP3775233A1/en not_active Withdrawn
- 2019-04-05 MX MX2020010477A patent/MX2020010477A/es unknown
- 2019-04-05 CA CA3096088A patent/CA3096088A1/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 |
---|---|
PH12020551641A1 (en) | 2021-07-26 |
BR112020020204A2 (pt) | 2021-01-19 |
US20190307900A1 (en) | 2019-10-10 |
JP2021520232A (ja) | 2021-08-19 |
TW202003052A (zh) | 2020-01-16 |
CA3096088A1 (en) | 2019-10-10 |
CN113056561A (zh) | 2021-06-29 |
CO2020013690A2 (es) | 2021-04-19 |
SG11202009759SA (en) | 2020-10-29 |
CL2020002561A1 (es) | 2021-04-23 |
MX2020010477A (es) | 2021-03-02 |
KR20210005040A (ko) | 2021-01-13 |
EA202092069A1 (ru) | 2021-03-12 |
IL277779A (en) | 2020-11-30 |
US20230149566A1 (en) | 2023-05-18 |
EP3775233A1 (en) | 2021-02-17 |
AU2019247864A1 (en) | 2020-10-22 |
MA52199A (fr) | 2021-02-17 |
RU2020132890A (ru) | 2022-05-06 |
JOP20200253A1 (ar) | 2020-10-04 |
WO2019195727A1 (en) | 2019-10-10 |
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