KR20210005040A - 황반이영양증을 치료하기 위한 조성물 및 방법 - Google Patents

황반이영양증을 치료하기 위한 조성물 및 방법 Download PDF

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KR20210005040A
KR20210005040A KR1020207031540A KR20207031540A KR20210005040A KR 20210005040 A KR20210005040 A KR 20210005040A KR 1020207031540 A KR1020207031540 A KR 1020207031540A KR 20207031540 A KR20207031540 A KR 20207031540A KR 20210005040 A KR20210005040 A KR 20210005040A
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South Korea
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sequence encoding
sequence
vector
composition
aav
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Korean (ko)
Inventor
데 라 카마라 크리스티나 마르티네스-페르난데스
로버트 매클라렌
그레고리 에스. 로빈슨
Original Assignee
옥스포드 유니버시티 이노베이션 리미티드
나이트스타엑스 리미티드
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Publication of KR20210005040A publication Critical patent/KR20210005040A/ko
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector 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)
KR1020207031540A 2018-04-05 2019-04-05 황반이영양증을 치료하기 위한 조성물 및 방법 Withdrawn KR20210005040A (ko)

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
KR20210005040A true KR20210005040A (ko) 2021-01-13

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Family Applications (1)

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KR1020207031540A Withdrawn KR20210005040A (ko) 2018-04-05 2019-04-05 황반이영양증을 치료하기 위한 조성물 및 방법

Country Status (21)

Country Link
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2307551T3 (pl) * 2008-06-18 2017-07-31 Oxford Biomedica (Uk) Limited Oczyszczanie wektorów retrowirusowych
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
AU2014359136B2 (en) * 2013-12-06 2020-06-25 Centre Hospitalier Universitaire De Montpellier 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
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

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Publication number Publication date
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
JP2021520232A (ja) 2021-08-19
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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PA0105 International application

Patent event date: 20201030

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination