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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
Application number
MX2020010477A
Other languages
English (en)
Inventor
Robert Maclaren
Gregory S Robinson
De La Camara Cristina Martinez-Fernandez
Original Assignee
Univ Oxford Innovation Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Oxford Innovation Ltd filed Critical Univ Oxford Innovation Ltd
Publication of MX2020010477A publication Critical patent/MX2020010477A/es

Links

Classifications

    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Ophthalmology & Optometry (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

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.
MX2020010477A 2018-04-05 2019-04-05 Composiciones y metodos para el tratamiento de la distrofia macular. MX2020010477A (es)

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 (5)

* Cited by examiner, † Cited by third party
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
AU2021226603A1 (en) * 2020-02-28 2022-10-27 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
WO2024064608A2 (en) * 2022-09-20 2024-03-28 Intergalactic Therapeutics, Inc. Best1 vectors and uses thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
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
US11098094B2 (en) * 2013-11-20 2021-08-24 Fondazione Telethon Artificial DNA-binding proteins and uses thereof
CN105940109A (zh) * 2013-12-06 2016-09-14 国立健康与医学研究所 用于在受试者的视网膜色素上皮中表达目的多核苷酸的方法和药物组合物
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

Also Published As

Publication number Publication date
WO2019195727A1 (en) 2019-10-10
TW202003052A (zh) 2020-01-16
CA3096088A1 (en) 2019-10-10
US20230149566A1 (en) 2023-05-18
CN113056561A (zh) 2021-06-29
AU2019247864A1 (en) 2020-10-22
IL277779A (en) 2020-11-30
MA52199A (fr) 2021-02-17
CO2020013690A2 (es) 2021-04-19
CL2020002561A1 (es) 2021-04-23
EA202092069A1 (ru) 2021-03-12
PE20210918A1 (es) 2021-05-19
BR112020020204A2 (pt) 2021-01-19
JOP20200253A1 (ar) 2020-10-04
RU2020132890A (ru) 2022-05-06
KR20210005040A (ko) 2021-01-13
US20190307900A1 (en) 2019-10-10
SG11202009759SA (en) 2020-10-29
EP3775233A1 (en) 2021-02-17
JP2021520232A (ja) 2021-08-19
PH12020551641A1 (en) 2021-07-26

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