PL3920912T3 - Leczenie ciliopatii przy użyciu inhibitorów syntazy glukozyloceramidowej (gcs) - Google Patents

Leczenie ciliopatii przy użyciu inhibitorów syntazy glukozyloceramidowej (gcs)

Info

Publication number
PL3920912T3
PL3920912T3 PL20709049.9T PL20709049T PL3920912T3 PL 3920912 T3 PL3920912 T3 PL 3920912T3 PL 20709049 T PL20709049 T PL 20709049T PL 3920912 T3 PL3920912 T3 PL 3920912T3
Authority
PL
Poland
Prior art keywords
ciliopathies
gcs
inhibitors
treatment
glucosylceramide synthase
Prior art date
Application number
PL20709049.9T
Other languages
English (en)
Inventor
Oxana Ibraghimov-Beskrovnaya
Nikolay O. Bukanov
Hervé HUSSON
Sarah E. MORENO
Original Assignee
Genzyme Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of PL3920912T3 publication Critical patent/PL3920912T3/pl

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/439Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • 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)
PL20709049.9T 2019-02-04 2020-02-04 Leczenie ciliopatii przy użyciu inhibitorów syntazy glukozyloceramidowej (gcs) PL3920912T3 (pl)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
PL3920912T3 true PL3920912T3 (pl) 2025-11-12

Family

ID=69740838

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20709049.9T PL3920912T3 (pl) 2019-02-04 2020-02-04 Leczenie ciliopatii przy użyciu inhibitorów syntazy glukozyloceramidowej (gcs)

Country Status (11)

Country Link
US (1) US20220110922A1 (pl)
EP (1) EP3920912B1 (pl)
JP (1) JP7633169B2 (pl)
CN (1) CN114040762A (pl)
AU (1) AU2020218185B2 (pl)
CA (1) CA3128043A1 (pl)
ES (1) ES3040555T3 (pl)
HU (1) HUE072626T2 (pl)
PL (1) PL3920912T3 (pl)
PT (1) PT3920912T (pl)
WO (1) WO2020163337A1 (pl)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
IL295255A (en) 2020-02-03 2022-10-01 Genzyme Corp Methods for treating neurological symptoms associated with lysosomal storage diseases
MX2023001014A (es) 2020-07-24 2023-03-01 Genzyme Corp Composiciones farmaceuticas que comprenden 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
WO2024116127A1 (en) 2022-12-01 2024-06-06 Genzyme Corporation Venglustat in combination with a strong or moderate inhibitor of cyp3a4
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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE640616A (pl) 1962-12-19
US3492397A (en) 1967-04-07 1970-01-27 Warner Lambert Pharmaceutical Sustained release dosage in the pellet form and process thereof
US4060598A (en) 1967-06-28 1977-11-29 Boehringer Mannheim G.M.B.H. Tablets coated with aqueous resin dispersions
US3538214A (en) 1969-04-22 1970-11-03 Merck & Co Inc Controlled release medicinal tablets
US4173626A (en) 1978-12-11 1979-11-06 Merck & Co., Inc. Sustained release indomethacin
LT3673906T (lt) 2011-03-18 2025-08-11 Genzyme Corporation Gliukozilceramido sintazės inhibitoriai
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
JO3713B1 (ar) 2013-03-15 2021-01-31 Genzyme Corp أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت
TW201642855A (zh) * 2015-03-10 2016-12-16 健臻公司 用於治療蛋白質病變之方法
JP6895963B2 (ja) * 2015-11-18 2021-06-30 ジェンザイム・コーポレーション 多発性嚢胞腎のバイオマーカーおよびその使用
US10588888B2 (en) * 2017-07-17 2020-03-17 Children's Hospital Medical Center CNS-accessible pharmacological chaperones for treatment of acid β-glucosidase-related disease states

Also Published As

Publication number Publication date
JP2022519131A (ja) 2022-03-18
PT3920912T (pt) 2025-09-09
CN114040762A (zh) 2022-02-11
EP3920912A1 (en) 2021-12-15
AU2020218185A1 (en) 2021-08-26
EP3920912B1 (en) 2025-06-11
ES3040555T3 (en) 2025-11-03
WO2020163337A1 (en) 2020-08-13
US20220110922A1 (en) 2022-04-14
CA3128043A1 (en) 2020-08-13
JP7633169B2 (ja) 2025-02-19
AU2020218185B2 (en) 2025-01-23
HUE072626T2 (hu) 2025-11-28

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