AU2020217659B2 - Methods for treating symptoms and disorders associated with lysosomal storage diseases - Google Patents

Methods for treating symptoms and disorders associated with lysosomal storage diseases Download PDF

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Publication number
AU2020217659B2
AU2020217659B2 AU2020217659A AU2020217659A AU2020217659B2 AU 2020217659 B2 AU2020217659 B2 AU 2020217659B2 AU 2020217659 A AU2020217659 A AU 2020217659A AU 2020217659 A AU2020217659 A AU 2020217659A AU 2020217659 B2 AU2020217659 B2 AU 2020217659B2
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compound
subject
halogen
alkyloxy
optionally substituted
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AU2020217659A
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AU2020217659A1 (en
Inventor
Nigel Patrick Somerville CRAWFORD
Tanya Zaremba FISCHER
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2020217659A 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases Active AU2020217659B2 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201962800996P 2019-02-04 2019-02-04
US62/800,996 2019-02-04
US201962851433P 2019-05-22 2019-05-22
US62/851,433 2019-05-22
US201962894167P 2019-08-30 2019-08-30
US62/894,167 2019-08-30
US201962937618P 2019-11-19 2019-11-19
US62/937,618 2019-11-19
US202062962647P 2020-01-17 2020-01-17
US62/962,647 2020-01-17
PCT/US2020/016441 WO2020163245A1 (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases

Publications (2)

Publication Number Publication Date
AU2020217659A1 AU2020217659A1 (en) 2021-08-26
AU2020217659B2 true AU2020217659B2 (en) 2025-04-17

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AU2020217659A Active AU2020217659B2 (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases
AU2020218496A Active AU2020218496B2 (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases

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AU2020218496A Active AU2020218496B2 (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases

Country Status (13)

Country Link
US (2) US20220016092A1 (https=)
EP (2) EP3920913A1 (https=)
JP (3) JP2022520747A (https=)
KR (2) KR20210123353A (https=)
CN (2) CN113645969A (https=)
AU (2) AU2020217659B2 (https=)
BR (2) BR112021015172A2 (https=)
CA (1) CA3128039A1 (https=)
IL (3) IL285304B2 (https=)
MX (2) MX2021009384A (https=)
SG (2) SG11202107842QA (https=)
TW (3) TW202045168A (https=)
WO (2) WO2020163245A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160207933A1 (en) * 2011-03-18 2016-07-21 Genzyme Corporation Glucosylceramide synthase inhibitors
US20180036295A1 (en) * 2015-03-10 2018-02-08 Genzyme Corporation Methods for treating proteinopathies

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE640616A (https=) 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
GB0400812D0 (en) 2004-01-14 2004-02-18 Celltech R&D Ltd Novel compounds
CA2840224C (en) * 2011-06-22 2019-08-13 The General Hospital Corporation Treatment of proteinopathies
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
EP3079695B1 (en) 2013-12-11 2021-03-17 Genzyme Corporation Glucosylceramide synthase inhibitors

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160207933A1 (en) * 2011-03-18 2016-07-21 Genzyme Corporation Glucosylceramide synthase inhibitors
US20180036295A1 (en) * 2015-03-10 2018-02-08 Genzyme Corporation Methods for treating proteinopathies

Also Published As

Publication number Publication date
CN113645969A (zh) 2021-11-12
TW202045167A (zh) 2020-12-16
JP2022520747A (ja) 2022-04-01
KR20210123352A (ko) 2021-10-13
CA3128039A1 (en) 2020-08-13
BR112021015172A2 (pt) 2021-09-28
JP2025072556A (ja) 2025-05-09
WO2020163244A1 (en) 2020-08-13
BR112021015099A2 (pt) 2021-10-05
TW202508582A (zh) 2025-03-01
WO2020163245A1 (en) 2020-08-13
IL285304B2 (en) 2025-10-01
IL285304A (en) 2021-09-30
SG11202107842QA (en) 2021-08-30
SG11202107844UA (en) 2021-08-30
AU2020218496A1 (en) 2021-08-26
JP7511567B2 (ja) 2024-07-05
IL285187A (en) 2021-09-30
TWI865485B (zh) 2024-12-11
CN113710249A (zh) 2021-11-26
CA3128041A1 (en) 2020-08-13
KR20210123353A (ko) 2021-10-13
IL313808A (en) 2024-08-01
US20220023273A1 (en) 2022-01-27
EP3920914A1 (en) 2021-12-15
US20220016092A1 (en) 2022-01-20
MX2021009383A (es) 2021-09-10
TW202045168A (zh) 2020-12-16
JP2022519274A (ja) 2022-03-22
EP3920913A1 (en) 2021-12-15
IL285304B1 (en) 2025-06-01
AU2020217659A1 (en) 2021-08-26
AU2020218496B2 (en) 2024-12-19
MX2021009384A (es) 2021-09-10

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