JP7211960B2 - 眼疾患の遺伝子治療 - Google Patents

眼疾患の遺伝子治療 Download PDF

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JP7211960B2
JP7211960B2 JP2019547457A JP2019547457A JP7211960B2 JP 7211960 B2 JP7211960 B2 JP 7211960B2 JP 2019547457 A JP2019547457 A JP 2019547457A JP 2019547457 A JP2019547457 A JP 2019547457A JP 7211960 B2 JP7211960 B2 JP 7211960B2
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lca5
aav
promoter
raav
sequence
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JP2020510433A5 (https=
JP2020510433A (ja
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ベネット，ジャン
ベニセリ，ジャネット
サン，ジャンウェイ
ソン，ジユン
ニコノフ，セルゲイ
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2019547457A 2017-03-01 2018-03-01 眼疾患の遺伝子治療 Active JP7211960B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023002799A JP7671790B2 (ja) 2017-03-01 2023-01-12 眼疾患の遺伝子治療
JP2025068834A JP2025114604A (ja) 2017-03-01 2025-04-18 眼疾患の遺伝子治療

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762465649P 2017-03-01 2017-03-01
US62/465,649 2017-03-01
US201762469642P 2017-03-10 2017-03-10
US62/469,642 2017-03-10
PCT/US2018/020470 WO2018160849A1 (en) 2017-03-01 2018-03-01 Gene therapy for ocular disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023002799A Division JP7671790B2 (ja) 2017-03-01 2023-01-12 眼疾患の遺伝子治療

Publications (3)

Publication Number Publication Date
JP2020510433A JP2020510433A (ja) 2020-04-09
JP2020510433A5 JP2020510433A5 (https=) 2021-03-18
JP7211960B2 true JP7211960B2 (ja) 2023-01-24

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JP2019547457A Active JP7211960B2 (ja) 2017-03-01 2018-03-01 眼疾患の遺伝子治療
JP2023002799A Active JP7671790B2 (ja) 2017-03-01 2023-01-12 眼疾患の遺伝子治療
JP2025068834A Pending JP2025114604A (ja) 2017-03-01 2025-04-18 眼疾患の遺伝子治療

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JP2023002799A Active JP7671790B2 (ja) 2017-03-01 2023-01-12 眼疾患の遺伝子治療
JP2025068834A Pending JP2025114604A (ja) 2017-03-01 2025-04-18 眼疾患の遺伝子治療

Country Status (11)

Country Link
US (2) US11564996B2 (https=)
EP (2) EP4656244A3 (https=)
JP (3) JP7211960B2 (https=)
KR (3) KR20230093072A (https=)
CN (2) CN110582572A (https=)
AU (2) AU2018228881B2 (https=)
BR (1) BR112019017327A2 (https=)
CA (1) CA3054136A1 (https=)
IL (2) IL314284A (https=)
RU (1) RU2019130004A (https=)
WO (1) WO2018160849A1 (https=)

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KR20240014102A (ko) * 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
JP7640549B2 (ja) * 2019-12-12 2025-03-05 ノバルティス アーゲー 注射デバイス及び注射溶液移し替えシステム
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20220387627A1 (en) * 2021-06-03 2022-12-08 Hope for Hasti Vectors and gene therapy for treating cornelia de lange syndrome

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Publication number Publication date
WO2018160849A8 (en) 2019-10-03
JP2025114604A (ja) 2025-08-05
AU2018228881B2 (en) 2024-01-25
CA3054136A1 (en) 2018-09-07
EP4656244A2 (en) 2025-12-03
CN110582572A (zh) 2019-12-17
AU2024202693A1 (en) 2024-05-16
IL314284A (en) 2024-09-01
US20190388561A1 (en) 2019-12-26
WO2018160849A1 (en) 2018-09-07
RU2019130004A3 (https=) 2021-07-05
KR102545070B1 (ko) 2023-06-19
EP3589738A1 (en) 2020-01-08
CN119752819A (zh) 2025-04-04
KR20240104196A (ko) 2024-07-04
EP3589738A4 (en) 2021-01-06
JP2023040219A (ja) 2023-03-22
JP7671790B2 (ja) 2025-05-02
EP4656244A3 (en) 2026-03-25
IL268946A (en) 2019-10-31
US20230233709A1 (en) 2023-07-27
BR112019017327A2 (pt) 2020-04-14
JP2020510433A (ja) 2020-04-09
KR20190125357A (ko) 2019-11-06
KR20230093072A (ko) 2023-06-26
US11564996B2 (en) 2023-01-31
RU2019130004A (ru) 2021-04-01
AU2018228881A1 (en) 2019-09-12

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