KR102636537B1 - 글리코겐 축적 질환 유형 iii에 대한 치료제 - Google Patents

글리코겐 축적 질환 유형 iii에 대한 치료제 Download PDF

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KR102636537B1
KR102636537B1 KR1020197036416A KR20197036416A KR102636537B1 KR 102636537 B1 KR102636537 B1 KR 102636537B1 KR 1020197036416 A KR1020197036416 A KR 1020197036416A KR 20197036416 A KR20197036416 A KR 20197036416A KR 102636537 B1 KR102636537 B1 KR 102636537B1
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polynucleotide
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KR20200014319A (ko
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기요시 다치카와
카를로스 구스타보 페레즈-가르시아
파드마나브 치부쿨라
하리 바스카란
크리스티안 더블유. 코보
숀 크리스토퍼 도허티
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울트라제닉스 파마수티컬 인코포레이티드
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    • C12N9/1051—Hexosyltransferases (2.4.1)
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    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
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    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
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    • 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)
    • C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)
    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
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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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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020197036416A 2017-05-31 2018-05-31 글리코겐 축적 질환 유형 iii에 대한 치료제 Active KR102636537B1 (ko)

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Application Number Priority Date Filing Date Title
US201762513350P 2017-05-31 2017-05-31
US62/513,350 2017-05-31
PCT/US2018/035477 WO2018222926A1 (en) 2017-05-31 2018-05-31 Therapeutics for glycogen storage disease type iii

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KR20200014319A KR20200014319A (ko) 2020-02-10
KR102636537B1 true KR102636537B1 (ko) 2024-02-15

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US (2) US11377643B2 (https=)
EP (1) EP3630964B1 (https=)
JP (2) JP7284101B2 (https=)
KR (1) KR102636537B1 (https=)
CN (1) CN110719954B (https=)
AR (1) AR112706A1 (https=)
AU (1) AU2018278315B2 (https=)
BR (1) BR112019025224A2 (https=)
CA (1) CA3063907A1 (https=)
CO (1) CO2019013332A2 (https=)
ES (1) ES3048960T3 (https=)
MX (1) MX2019014412A (https=)
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BR112019025224A2 (pt) 2020-12-08
CA3063907A1 (en) 2018-12-06
US11377643B2 (en) 2022-07-05
AU2018278315B2 (en) 2024-01-18
EP3630964C0 (en) 2025-08-13
US20220340886A1 (en) 2022-10-27
JP2023075248A (ja) 2023-05-30
JP7284101B2 (ja) 2023-05-30
CN110719954A (zh) 2020-01-21
TWI794237B (zh) 2023-03-01
TW201903151A (zh) 2019-01-16
MX2019014412A (es) 2020-02-10
AU2018278315A1 (en) 2019-12-12
EP3630964A4 (en) 2021-03-03
WO2018222926A1 (en) 2018-12-06
AR112706A1 (es) 2019-12-04
EP3630964A1 (en) 2020-04-08
ES3048960T3 (en) 2025-12-12
JP2020522244A (ja) 2020-07-30
CN110719954B (zh) 2023-12-26
US20200149017A1 (en) 2020-05-14
CO2019013332A2 (es) 2020-02-18
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