JP2021520232A - 黄斑ジストロフィーを処置するための組成物及び方法 - Google Patents

黄斑ジストロフィーを処置するための組成物及び方法 Download PDF

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JP2021520232A
JP2021520232A JP2021504131A JP2021504131A JP2021520232A JP 2021520232 A JP2021520232 A JP 2021520232A JP 2021504131 A JP2021504131 A JP 2021504131A JP 2021504131 A JP2021504131 A JP 2021504131A JP 2021520232 A JP2021520232 A JP 2021520232A
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sequence encoding
sequence
cell
best1
aav
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JPWO2019195727A5 (https=
JP2021520232A5 (https=
Inventor
グレゴリー エス． ロビンソン，
グレゴリー エス． ロビンソン，
デ ラ カマラ， クリスティーナ マルティネス−フェルナンデス
デ ラ カマラ， クリスティーナ マルティネス−フェルナンデス
ロバート マクラーレン，
ロバート マクラーレン，
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Oxford University Innovation Ltd
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Oxford University Innovation Ltd
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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
    • 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
    • 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)
JP2021504131A 2018-04-05 2019-04-05 黄斑ジストロフィーを処置するための組成物及び方法 Withdrawn JP2021520232A (ja)

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 (3)

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JP2021520232A true JP2021520232A (ja) 2021-08-19
JPWO2019195727A5 JPWO2019195727A5 (https=) 2022-04-12
JP2021520232A5 JP2021520232A5 (https=) 2022-04-12

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JP2021504131A Withdrawn JP2021520232A (ja) 2018-04-05 2019-04-05 黄斑ジストロフィーを処置するための組成物及び方法

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3870148A4 (en) * 2018-10-25 2022-11-09 Takeda Pharmaceutical Company Limited AAV TRIPLE PLASMID SYSTEM
US20220089670A1 (en) * 2018-12-28 2022-03-24 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
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011524887A (ja) * 2008-06-18 2011-09-08 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルス精製法
JP2017500060A (ja) * 2013-12-06 2017-01-05 インセルム（インスティチュート ナショナル デ ラ サンテ エ デ ラ リシェルシェ メディカル） 対象の網膜色素上皮において目的のポリヌクレオチドを発現させるための方法および医薬組成物
JP2017500870A (ja) * 2013-12-20 2017-01-12 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルスベクター産生系

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP3071592B1 (en) * 2013-11-20 2021-01-06 Fondazione Telethon Artificial dna-binding proteins and uses thereof
RS63416B1 (sr) * 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
WO2017083722A1 (en) * 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011524887A (ja) * 2008-06-18 2011-09-08 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルス精製法
JP2017500060A (ja) * 2013-12-06 2017-01-05 インセルム（インスティチュート ナショナル デ ラ サンテ エ デ ラ リシェルシェ メディカル） 対象の網膜色素上皮において目的のポリヌクレオチドを発現させるための方法および医薬組成物
JP2017500870A (ja) * 2013-12-20 2017-01-12 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルスベクター産生系

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GUZIEWICZ KE. ET AL.: "Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Depe", PLOS ONE, 2013, VOL. 8, NO. 10, E75666, JPN6023012097, ISSN: 0005024073 *

Also Published As

Publication number Publication date
KR20210005040A (ko) 2021-01-13
PE20210918A1 (es) 2021-05-19
EA202092069A1 (ru) 2021-03-12
MA52199A (fr) 2021-02-17
MX2020010477A (es) 2021-03-02
US20230149566A1 (en) 2023-05-18
CN113056561A (zh) 2021-06-29
JOP20200253A1 (ar) 2020-10-04
CA3096088A1 (en) 2019-10-10
IL277779A (en) 2020-11-30
PH12020551641A1 (en) 2021-07-26
RU2020132890A (ru) 2022-05-06
CO2020013690A2 (es) 2021-04-19
BR112020020204A2 (pt) 2021-01-19
CL2020002561A1 (es) 2021-04-23
AU2019247864A1 (en) 2020-10-22
US20190307900A1 (en) 2019-10-10
EP3775233A1 (en) 2021-02-17
SG11202009759SA (en) 2020-10-29
WO2019195727A1 (en) 2019-10-10
TW202003052A (zh) 2020-01-16

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