JP7284101B2 - 糖原病ｉｉｉ型のための治療薬 - Google Patents

糖原病ｉｉｉ型のための治療薬 Download PDF

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JP7284101B2
JP7284101B2 JP2019565348A JP2019565348A JP7284101B2 JP 7284101 B2 JP7284101 B2 JP 7284101B2 JP 2019565348 A JP2019565348 A JP 2019565348A JP 2019565348 A JP2019565348 A JP 2019565348A JP 7284101 B2 JP7284101 B2 JP 7284101B2
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agl
molecules
polynucleotide
molecule
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JP2020522244A5 (2
JP2020522244A (ja
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キヨシ タチカワ
カルロス グスタヴォ ペレス－ガルシア
パドマナブ チブクラ
ハリ バースカラン
クリスチャン ダブリュー． コボー
ショーン クリストファー ドアティ
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Ultragenyx Pharmaceutical Inc
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    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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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
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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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    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01025—4-Alpha-glucanotransferase (2.4.1.25)
    • 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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    • 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
    • A61K48/0008—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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • 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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    • 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)
    • C12Y302/01033—Amylo-alpha-1,6-glucosidase (3.2.1.33)

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  • Gastroenterology & Hepatology (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)
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  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2019565348A 2017-05-31 2018-05-31 糖原病ｉｉｉ型のための治療薬 Active JP7284101B2 (ja)

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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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US (2) US11377643B2 (2)
EP (1) EP3630964B1 (2)
JP (2) JP7284101B2 (2)
KR (1) KR102636537B1 (2)
CN (1) CN110719954B (2)
AR (1) AR112706A1 (2)
AU (1) AU2018278315B2 (2)
BR (1) BR112019025224A2 (2)
CA (1) CA3063907A1 (2)
CO (1) CO2019013332A2 (2)
ES (1) ES3048960T3 (2)
MX (1) MX2019014412A (2)
TW (1) TWI794237B (2)
WO (1) WO2018222926A1 (2)

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AU2018278315A1 (en) 2019-12-12
CN110719954A (zh) 2020-01-21
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WO2018222926A1 (en) 2018-12-06
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US11377643B2 (en) 2022-07-05
US20200149017A1 (en) 2020-05-14
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MX2019014412A (es) 2020-02-10
CN110719954B (zh) 2023-12-26
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BR112019025224A2 (pt) 2020-12-08
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EP3630964A1 (en) 2020-04-08
JP2023075248A (ja) 2023-05-30
CO2019013332A2 (es) 2020-02-18
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