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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    • C12N9/14—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)
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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
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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
    • C12N2310/30—Chemical structure
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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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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • 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 (enExample)
EP (1) EP3630964B1 (enExample)
JP (2) JP7284101B2 (enExample)
KR (1) KR102636537B1 (enExample)
CN (1) CN110719954B (enExample)
AR (1) AR112706A1 (enExample)
AU (1) AU2018278315B2 (enExample)
BR (1) BR112019025224A2 (enExample)
CA (1) CA3063907A1 (enExample)
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BR112019025224A2 (pt) 2020-12-08
AU2018278315A1 (en) 2019-12-12
ES3048960T3 (en) 2025-12-12
EP3630964A4 (en) 2021-03-03
US11377643B2 (en) 2022-07-05
US20220340886A1 (en) 2022-10-27
WO2018222926A1 (en) 2018-12-06
TWI794237B (zh) 2023-03-01
CA3063907A1 (en) 2018-12-06
EP3630964C0 (en) 2025-08-13
AU2018278315B2 (en) 2024-01-18
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JP2020522244A (ja) 2020-07-30
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