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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JP2020522244A (ja
JP2020522244A5 (enExample
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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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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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    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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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
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    • 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
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    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2451—Glucanases acting on alpha-1,6-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • 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
    • 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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    • 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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  • 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)
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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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CN (1) CN110719954B (enExample)
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WO2010005565A2 (en) 2008-07-08 2010-01-14 Duke University Method of treating glycogen storage disease
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KR20200014319A (ko) 2020-02-10
CN110719954A (zh) 2020-01-21
EP3630964A1 (en) 2020-04-08
KR102636537B1 (ko) 2024-02-15
US11377643B2 (en) 2022-07-05
US20220340886A1 (en) 2022-10-27
EP3630964B1 (en) 2025-08-13
AU2018278315A1 (en) 2019-12-12
AU2018278315B2 (en) 2024-01-18
CO2019013332A2 (es) 2020-02-18
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WO2018222926A1 (en) 2018-12-06
EP3630964C0 (en) 2025-08-13
BR112019025224A2 (pt) 2020-12-08
CN110719954B (zh) 2023-12-26
CA3063907A1 (en) 2018-12-06
TW201903151A (zh) 2019-01-16
US20200149017A1 (en) 2020-05-14
AR112706A1 (es) 2019-12-04
MX2019014412A (es) 2020-02-10
JP2020522244A (ja) 2020-07-30
JP2023075248A (ja) 2023-05-30
EP3630964A4 (en) 2021-03-03
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