KR102763312B1 - 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법 - Google Patents

치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법 Download PDF

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KR102763312B1
KR102763312B1 KR1020207014057A KR20207014057A KR102763312B1 KR 102763312 B1 KR102763312 B1 KR 102763312B1 KR 1020207014057 A KR1020207014057 A KR 1020207014057A KR 20207014057 A KR20207014057 A KR 20207014057A KR 102763312 B1 KR102763312 B1 KR 102763312B1
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polynucleotide
leu
gly
ala
ser
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KR20200103634A (ko
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드니즈 달카라
아넹 카부
조제-알랭 사엘
띠에리 르베이야르
졍 뒤에벨
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인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔)
쏘흐본느 유니베흐시테
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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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/125—Picornaviridae, e.g. calicivirus
    • 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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
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  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020207014057A 2017-10-20 2018-10-22 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법 Active KR102763312B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17306430.4 2017-10-20
EP17306430 2017-10-20
PCT/EP2018/078856 WO2019077159A1 (en) 2017-10-20 2018-10-22 METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR

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KR20200103634A KR20200103634A (ko) 2020-09-02
KR102763312B1 true KR102763312B1 (ko) 2025-02-07

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Country Status (6)

Country Link
US (1) US11723988B2 (https=)
EP (1) EP3697448A1 (https=)
JP (1) JP7420710B2 (https=)
KR (1) KR102763312B1 (https=)
CN (1) CN111867635A (https=)
WO (1) WO2019077159A1 (https=)

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EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
CN110709095B (zh) 2017-04-05 2024-05-03 马萨诸塞大学 小基因治疗
WO2020236815A1 (en) 2019-05-20 2020-11-26 University Of Massachusetts Minigene therapy
US20230159609A1 (en) * 2020-04-10 2023-05-25 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
CA3195696A1 (en) * 2020-09-17 2022-03-24 Restore Vision Inc. Composition for treating or preventing diseases, disorders, or conditions associated with endoplasmic reticulum stress or all-trans-retinal, protecting retinal thickness, or suppressing reduction in retinal thickness or progress of reduction in retinal thickness
CN113121655B (zh) * 2021-04-19 2021-11-19 上海信致医药科技有限公司 眼部和肌肉特异靶向型腺相关病毒载体及其应用
WO2022223644A2 (en) 2021-04-20 2022-10-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for treating retinal degenerative disorders
CA3221439A1 (en) * 2021-05-26 2022-12-01 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
IL320351A (en) 2022-10-20 2025-06-01 Sparingvision Compositions and methods for treating retinal degenerative disorders
WO2024110770A1 (en) * 2022-11-22 2024-05-30 INSERM (Institut National de la Santé et de la Recherche Médicale) A new promoter for retinal pigment epithelium (rpe) targeted gene therapy
WO2024182558A1 (en) * 2023-02-28 2024-09-06 The Saban Research Institute; Children's Hospital Los Angeles Fluorescent cone reporter ips cells, retinal organoids and uses thereof
CN117247434B (zh) * 2023-11-10 2024-02-02 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
CN119350454B (zh) * 2024-12-27 2025-03-25 上海朗昇生物科技有限公司 衣壳重排型腺相关病毒载体及其用途

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Also Published As

Publication number Publication date
KR20200103634A (ko) 2020-09-02
JP2021500030A (ja) 2021-01-07
CN111867635A (zh) 2020-10-30
US20210268125A1 (en) 2021-09-02
WO2019077159A1 (en) 2019-04-25
US11723988B2 (en) 2023-08-15
JP7420710B2 (ja) 2024-01-23
EP3697448A1 (en) 2020-08-26

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