AU2020218185B2 - Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS) - Google Patents

Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS) Download PDF

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AU2020218185B2
AU2020218185B2 AU2020218185A AU2020218185A AU2020218185B2 AU 2020218185 B2 AU2020218185 B2 AU 2020218185B2 AU 2020218185 A AU2020218185 A AU 2020218185A AU 2020218185 A AU2020218185 A AU 2020218185A AU 2020218185 B2 AU2020218185 B2 AU 2020218185B2
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compound
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syndrome
ciliopathy
bbs
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AU2020218185A1 (en
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Nikolay O. Bukanov
Hervé HUSSON
Oxana Ibraghimov-Beskrovnaya
Sarah E. MORENO
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Genzyme Corp
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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)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2020218185A 2019-02-04 2020-02-04 Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS) Active AU2020218185B2 (en)

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)

Publications (2)

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AU2020218185A1 AU2020218185A1 (en) 2021-08-26
AU2020218185B2 true AU2020218185B2 (en) 2025-01-23

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AU2020218185A Active AU2020218185B2 (en) 2019-02-04 2020-02-04 Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS)

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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=)
PT (1) PT3920912T (https=)
WO (1) WO2020163337A1 (https=)

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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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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
JP7633169B2 (ja) 2025-02-19
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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