RU2020132890A - Композиции и способы лечения макулярной дистрофии - Google Patents

Композиции и способы лечения макулярной дистрофии Download PDF

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Publication number
RU2020132890A
RU2020132890A RU2020132890A RU2020132890A RU2020132890A RU 2020132890 A RU2020132890 A RU 2020132890A RU 2020132890 A RU2020132890 A RU 2020132890A RU 2020132890 A RU2020132890 A RU 2020132890A RU 2020132890 A RU2020132890 A RU 2020132890A
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RU2020132890A
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Кристина Мартинес-Фернандес Де Ла Камара
Роберт МАКЛАРЕН
Грегори С. Робинсон
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Оксфорд Юниверсити Инновейшн Лимитед
Найтстаркс Лимитед
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Publication of RU2020132890A publication Critical patent/RU2020132890A/ru

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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
    • 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

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  • 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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Manufacturing & Machinery (AREA)
  • Dermatology (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)
RU2020132890A 2018-04-05 2019-04-05 Композиции и способы лечения макулярной дистрофии RU2020132890A (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862653131P 2018-04-05 2018-04-05
US62/653,131 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
RU2020132890A true RU2020132890A (ru) 2022-05-06

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
RU2020132890A RU2020132890A (ru) 2018-04-05 2019-04-05 Композиции и способы лечения макулярной дистрофии

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US (2) US20190307900A1 (enExample)
EP (1) EP3775233A1 (enExample)
JP (1) JP2021520232A (enExample)
KR (1) KR20210005040A (enExample)
CN (1) CN113056561A (enExample)
AU (1) AU2019247864A1 (enExample)
BR (1) BR112020020204A2 (enExample)
CA (1) CA3096088A1 (enExample)
CL (1) CL2020002561A1 (enExample)
CO (1) CO2020013690A2 (enExample)
EA (1) EA202092069A1 (enExample)
IL (1) IL277779A (enExample)
JO (1) JOP20200253A1 (enExample)
MA (1) MA52199A (enExample)
MX (1) MX2020010477A (enExample)
PE (1) PE20210918A1 (enExample)
PH (1) PH12020551641A1 (enExample)
RU (1) RU2020132890A (enExample)
SG (1) SG11202009759SA (enExample)
TW (1) TW202003052A (enExample)
WO (1) WO2019195727A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020047476A1 (en) * 2018-08-31 2020-03-05 University Of Florida Research Foundation, Incorporated Adeno-associated viral vectors for the treatment of best disease
EP3870148A4 (en) 2018-10-25 2022-11-09 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
BR112022017182A2 (pt) * 2020-02-28 2022-11-08 Univ Pennsylvania Tratamento de bestrofinopatias autossômicas recessivas e métodos para avaliação das mesmas
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
WO2009153563A1 (en) * 2008-06-18 2009-12-23 Oxford Biomedica (Uk) Limited Virus purification
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
CA2930282C (en) * 2013-11-20 2024-09-10 Universita Degli Studi Di Napoli Federico Ii Artificial DNA-Binding Proteins and Their Uses
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
CA2979120A1 (en) * 2015-03-03 2016-09-09 Fondazione Telethon Multiple aav vector system for expressing genes over 5 kilobases, comprising at least one degradation signal and uses thereof
EP3374494A4 (en) * 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE THEREOF FOR GENE THERAPY

Also Published As

Publication number Publication date
EP3775233A1 (en) 2021-02-17
AU2019247864A1 (en) 2020-10-22
WO2019195727A1 (en) 2019-10-10
MA52199A (fr) 2021-02-17
BR112020020204A2 (pt) 2021-01-19
PE20210918A1 (es) 2021-05-19
US20190307900A1 (en) 2019-10-10
JP2021520232A (ja) 2021-08-19
IL277779A (en) 2020-11-30
PH12020551641A1 (en) 2021-07-26
US20230149566A1 (en) 2023-05-18
MX2020010477A (es) 2021-03-02
JOP20200253A1 (ar) 2020-10-04
CL2020002561A1 (es) 2021-04-23
SG11202009759SA (en) 2020-10-29
CN113056561A (zh) 2021-06-29
KR20210005040A (ko) 2021-01-13
TW202003052A (zh) 2020-01-16
CA3096088A1 (en) 2019-10-10
CO2020013690A2 (es) 2021-04-19
EA202092069A1 (ru) 2021-03-12

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