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EA202091352A1 - Генотерапия мукополисахаридоза iiib - Google Patents

Генотерапия мукополисахаридоза iiib

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Publication number
EA202091352A1
EA202091352A1 EA202091352A EA202091352A EA202091352A1 EA 202091352 A1 EA202091352 A1 EA 202091352A1 EA 202091352 A EA202091352 A EA 202091352A EA 202091352 A EA202091352 A EA 202091352A EA 202091352 A1 EA202091352 A1 EA 202091352A1
Authority
EA
Eurasian Patent Office
Prior art keywords
aav
hnaglu
itr
raav
vector genome
Prior art date
Application number
EA202091352A
Other languages
English (en)
Inventor
Джеймс М. Уилсон
Кристиан Хиндерер
Жюльетт Ордо
Original Assignee
Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания
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Application filed by Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания filed Critical Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания
Publication of EA202091352A1 publication Critical patent/EA202091352A1/ru

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    • 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
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    • C12N9/14—Hydrolases (3)
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • 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
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04045—N-Acetylglucosamine-1-phosphodiester alpha-N-acetylglucosaminidase (3.1.4.45)
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
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  • Physics & Mathematics (AREA)
  • Mycology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

В данной заявке предложен рекомбинантный AAV (rAAV), содержащий капсид AAV и упакованный в нем геном вектора, где геном вектора содержит AAV 5' инвертированный концевой повтор (ITR), полученную способом генной инженерии последовательность нуклеиновой кислоты, кодирующую функциональную человеческую N-ацетил-альфа-глюкозаминидазу (hNAGLU), регуляторную последовательность, которая направляет экспрессию hNAGLU в клетке-мишени, и AAV 3' ITR. Также представлена фармацевтическая композиция, содержащая rAAV согласно настоящему описанию в буферной смеси, и способ лечения человеческого субъекта, у которого диагностирован MPS IIIB.
EA202091352A 2017-11-30 2018-11-29 Генотерапия мукополисахаридоза iiib EA202091352A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762593090P 2017-11-30 2017-11-30
PCT/US2018/063166 WO2019108856A1 (en) 2017-11-30 2018-11-29 Gene therapy for mucopolysaccharidosis iiib

Publications (1)

Publication Number Publication Date
EA202091352A1 true EA202091352A1 (ru) 2020-08-27

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EA202091352A EA202091352A1 (ru) 2017-11-30 2018-11-29 Генотерапия мукополисахаридоза iiib

Country Status (14)

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US (2) US11723989B2 (ru)
EP (1) EP3717652A4 (ru)
JP (1) JP7384797B2 (ru)
KR (1) KR20200104864A (ru)
CN (1) CN111989396A (ru)
AU (1) AU2018375163A1 (ru)
BR (1) BR112020010977A2 (ru)
CA (1) CA3083761A1 (ru)
CL (1) CL2020001428A1 (ru)
EA (1) EA202091352A1 (ru)
IL (1) IL274972A (ru)
MX (1) MX2020005673A (ru)
SG (1) SG11202004991SA (ru)
WO (1) WO2019108856A1 (ru)

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WO2021194915A1 (en) * 2020-03-23 2021-09-30 The University Of North Carolina At Chapel Hill Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
WO2021231491A1 (en) * 2020-05-11 2021-11-18 University Of Florida Research Foundation, Incorporated Disease correction by delivery of aav8 vectors expressing codon optimized naglu
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MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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US11723989B2 (en) 2023-08-15
CL2020001428A1 (es) 2020-10-23
CN111989396A (zh) 2020-11-24
WO2019108856A1 (en) 2019-06-06
SG11202004991SA (en) 2020-06-29
US20240009327A1 (en) 2024-01-11
IL274972A (en) 2020-07-30
JP7384797B2 (ja) 2023-11-21
EP3717652A1 (en) 2020-10-07
CA3083761A1 (en) 2019-06-06
MX2020005673A (es) 2020-12-03
JP2021507687A (ja) 2021-02-25
EP3717652A4 (en) 2021-11-24
BR112020010977A2 (pt) 2020-11-17
KR20200104864A (ko) 2020-09-04
US20200289675A1 (en) 2020-09-17
AU2018375163A1 (en) 2020-06-11

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