KR20220105158A - 이식유전자 발현의 drg-특이적 감소를 위한 조성물 - Google Patents

이식유전자 발현의 drg-특이적 감소를 위한 조성물 Download PDF

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KR20220105158A
KR20220105158A KR1020227016895A KR20227016895A KR20220105158A KR 20220105158 A KR20220105158 A KR 20220105158A KR 1020227016895 A KR1020227016895 A KR 1020227016895A KR 20227016895 A KR20227016895 A KR 20227016895A KR 20220105158 A KR20220105158 A KR 20220105158A
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South Korea
Prior art keywords
sequence
seq
hidua
nucleotides
expression cassette
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Pending
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KR1020227016895A
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English (en)
Korean (ko)
Inventor
줄리에트 호르도
제임스 엠. 윌슨
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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Priority claimed from PCT/US2019/067872 external-priority patent/WO2020132455A1/en
Application filed by 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 filed Critical 더 트러스티스 오브 더 유니버시티 오브 펜실베니아
Publication of KR20220105158A publication Critical patent/KR20220105158A/ko
Pending legal-status Critical Current

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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
    • C12N15/861—Adenoviral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • 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

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • General Preparation And Processing Of Foods (AREA)
  • Coloring Foods And Improving Nutritive Qualities (AREA)
KR1020227016895A 2019-10-23 2020-10-22 이식유전자 발현의 drg-특이적 감소를 위한 조성물 Pending KR20220105158A (ko)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US201962924970P 2019-10-23 2019-10-23
US62/924,970 2019-10-23
US201962934915P 2019-11-13 2019-11-13
US62/934,915 2019-11-13
USPCT/US2019/067872 2019-12-20
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression
US202062972404P 2020-02-10 2020-02-10
US62/972,404 2020-02-10
US202063005894P 2020-04-06 2020-04-06
US63/005,894 2020-04-06
US202063023602P 2020-05-12 2020-05-12
US63/023,602 2020-05-12
US202063038514P 2020-06-12 2020-06-12
US63/038,514 2020-06-12
US202063043600P 2020-06-24 2020-06-24
US63/043,600 2020-06-24
PCT/US2020/056881 WO2021081217A1 (en) 2019-10-23 2020-10-22 Compositions for drg-specific reduction of transgene expression

Publications (1)

Publication Number Publication Date
KR20220105158A true KR20220105158A (ko) 2022-07-26

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227016895A Pending KR20220105158A (ko) 2019-10-23 2020-10-22 이식유전자 발현의 drg-특이적 감소를 위한 조성물

Country Status (8)

Country Link
US (1) US20220389457A1 (https=)
EP (1) EP4048785A4 (https=)
JP (1) JP7697943B2 (https=)
KR (1) KR20220105158A (https=)
AU (1) AU2020369570A1 (https=)
CA (1) CA3155154A1 (https=)
IL (1) IL292372A (https=)
WO (1) WO2021081217A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230304034A1 (en) * 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
BR112023003310A2 (pt) * 2020-08-24 2023-05-02 Univ Pennsylvania Vetores virais que codificam fusões de agonistas do receptor de glp-1 e usos dos mesmos no tratamento de doenças metabólicas
AU2022369293A1 (en) * 2021-10-18 2024-05-16 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of cdkl5 deficiency disorder (cdd)
CA3236188A1 (en) * 2021-10-27 2023-05-04 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i
EP4551253A1 (en) * 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
CN116064593B (zh) * 2023-02-09 2024-05-14 四川大学 一种毛白杨pgag基因及其应用
US20250276095A1 (en) * 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
HK1256341A1 (zh) * 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
EP3411488A1 (en) * 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
KR20180109945A (ko) * 2016-02-22 2018-10-08 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Ｍｐｓ ｉ－ 연관 실명의 치료를 위한 ａａｖ－ｉｄｕａ 벡터
MA50016A (fr) * 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
SG11202002276VA (en) * 2017-09-20 2020-04-29 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof

Also Published As

Publication number Publication date
JP2022553406A (ja) 2022-12-22
JP7697943B2 (ja) 2025-06-24
IL292372A (en) 2022-06-01
EP4048785A1 (en) 2022-08-31
US20220389457A1 (en) 2022-12-08
EP4048785A4 (en) 2024-03-27
WO2021081217A1 (en) 2021-04-29
AU2020369570A1 (en) 2022-05-12
CA3155154A1 (en) 2021-04-29

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