IL285304B2 - Methods for treating symptoms and disorders associated with lysosomal storage diseases - Google Patents

Methods for treating symptoms and disorders associated with lysosomal storage diseases

Info

Publication number
IL285304B2
IL285304B2 IL285304A IL28530421A IL285304B2 IL 285304 B2 IL285304 B2 IL 285304B2 IL 285304 A IL285304 A IL 285304A IL 28530421 A IL28530421 A IL 28530421A IL 285304 B2 IL285304 B2 IL 285304B2
Authority
IL
Israel
Prior art keywords
compound
subject
disease
tmt
quinuclidin
Prior art date
Application number
IL285304A
Other languages
English (en)
Hebrew (he)
Other versions
IL285304A (en
IL285304B1 (en
Original Assignee
Genzyme Corp
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 Corp filed Critical Genzyme Corp
Publication of IL285304A publication Critical patent/IL285304A/en
Publication of IL285304B1 publication Critical patent/IL285304B1/en
Publication of IL285304B2 publication Critical patent/IL285304B2/en

Links

Classifications

    • 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

Landscapes

  • 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)
IL285304A 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases IL285304B2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962800996P 2019-02-04 2019-02-04
US201962851433P 2019-05-22 2019-05-22
US201962894167P 2019-08-30 2019-08-30
US201962937618P 2019-11-19 2019-11-19
US202062962647P 2020-01-17 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 (3)

Publication Number Publication Date
IL285304A IL285304A (en) 2021-09-30
IL285304B1 IL285304B1 (en) 2025-06-01
IL285304B2 true IL285304B2 (en) 2025-10-01

Family

ID=69743942

Family Applications (3)

Application Number Title Priority Date Filing Date
IL285304A IL285304B2 (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases
IL313808A IL313808A (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases
IL285187A IL285187A (en) 2019-02-04 2021-07-28 Methods for treating symptoms and disorders associated with lysosomal storage diseases

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL313808A IL313808A (en) 2019-02-04 2020-02-03 Methods for treating symptoms and disorders associated with lysosomal storage diseases
IL285187A IL285187A (en) 2019-02-04 2021-07-28 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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
A. MEHTA ET AL:, FABRY DISEASE: A REVIEW OF CURRENT MANAGEMENT STRATEGIES, 1 September 2010 (2010-09-01) *

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
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
AU2020217659B2 (en) 2025-04-17
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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