JP7633169B2 - グルコシルセラミドシンターゼ（ｇｃｓ）阻害剤を使用した繊毛病の処置 - Google Patents

グルコシルセラミドシンターゼ（ｇｃｓ）阻害剤を使用した繊毛病の処置 Download PDF

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JP7633169B2
JP7633169B2 JP2021545384A JP2021545384A JP7633169B2 JP 7633169 B2 JP7633169 B2 JP 7633169B2 JP 2021545384 A JP2021545384 A JP 2021545384A JP 2021545384 A JP2021545384 A JP 2021545384A JP 7633169 B2 JP7633169 B2 JP 7633169B2
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JP2022519131A (ja
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オクサナ・イブラギモフ－ベスクロフナヤ
ニコライ・オー・ブカノフ
エルヴェ・ユソン
サラ・イー・モレノ
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Genzyme Corp
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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/439—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring forming part of a bridged ring system, e.g. quinuclidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021545384A 2019-02-04 2020-02-04 グルコシルセラミドシンターゼ（ｇｃｓ）阻害剤を使用した繊毛病の処置 Active JP7633169B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962800993P 2019-02-04 2019-02-04
US62/800,993 2019-02-04
US201962851430P 2019-05-22 2019-05-22
US62/851,430 2019-05-22
PCT/US2020/016588 WO2020163337A1 (en) 2019-02-04 2020-02-04 Treatment of ciliopathies using inhibitors of glucosylceramide synthase (gcs)

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JP2022519131A JP2022519131A (ja) 2022-03-18
JP2022519131A5 JP2022519131A5 (https=) 2023-02-10
JP7633169B2 true JP7633169B2 (ja) 2025-02-19

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US (1) US12527776B2 (https=)
EP (1) EP3920912B1 (https=)
JP (1) JP7633169B2 (https=)
CN (1) CN114040762A (https=)
AU (1) AU2020218185B2 (https=)
ES (1) ES3040555T3 (https=)
HU (1) HUE072626T2 (https=)
PL (1) PL3920912T3 (https=)
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WO (1) WO2020163337A1 (https=)

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CN114040762A (zh) 2019-02-04 2022-02-11 建新公司 使用葡糖神经酰胺合酶(gcs)的抑制剂治疗纤毛疾病
JP2023512366A (ja) 2020-02-03 2023-03-27 ジェンザイム・コーポレーション リソソーム蓄積性疾患と関連する神経学的症状を処置するための方法
AU2021311131B2 (en) 2020-07-24 2026-02-19 Genzyme Corporation Pharmaceutical compositions comprising venglustat
WO2022169004A1 (ko) * 2021-02-05 2022-08-11 연세대학교 산학협력단 신규한 fabp4 억제제를 유효성분으로 포함하는 섬모형성 촉진용 조성물
EP4201403A1 (en) 2021-12-21 2023-06-28 Som Innovation Biotech, S.L. Compounds tirapazamine and quazinone for use in the treatment of gm2 gangliosidoses
CN115350278A (zh) * 2022-07-11 2022-11-18 清华大学 提高细胞内鞘脂类物质含量的试剂在制备纤毛异常相关疾病药物中的用途
JP2025541739A (ja) 2022-12-01 2025-12-23 ジェンザイム・コーポレーション Ｃｙｐ３ａ４の強力な又は中程度の阻害剤と組み合わせたベングルスタット
WO2025178019A1 (ja) * 2024-02-19 2025-08-28 国立大学法人大阪大学 繊毛病治療用医薬組成物
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

Citations (1)

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JP2015527406A (ja) 2012-09-11 2015-09-17 ジェンザイム・コーポレーション グルコシルセラミド合成酵素阻害剤

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EP3920912B1 (en) 2025-06-11
WO2020163337A1 (en) 2020-08-13
US20220110922A1 (en) 2022-04-14
PL3920912T3 (pl) 2025-11-12
AU2020218185A1 (en) 2021-08-26
AU2020218185B2 (en) 2025-01-23
CA3128043A1 (en) 2020-08-13
EP3920912A1 (en) 2021-12-15
JP2022519131A (ja) 2022-03-18
HUE072626T2 (hu) 2025-11-28
US12527776B2 (en) 2026-01-20
CN114040762A (zh) 2022-02-11
PT3920912T (pt) 2025-09-09
ES3040555T3 (en) 2025-11-03

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