CN113645969A - 用于治疗与溶酶体贮积症相关的症状和病症的方法 - Google Patents

用于治疗与溶酶体贮积症相关的症状和病症的方法 Download PDF

Info

Publication number
CN113645969A
CN113645969A CN202080025346.0A CN202080025346A CN113645969A CN 113645969 A CN113645969 A CN 113645969A CN 202080025346 A CN202080025346 A CN 202080025346A CN 113645969 A CN113645969 A CN 113645969A
Authority
CN
China
Prior art keywords
compound
subject
alkoxy
disease
halogen
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202080025346.0A
Other languages
English (en)
Chinese (zh)
Inventor
N·P·S·克劳福德
T·Z·菲舍尔
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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 CN113645969A publication Critical patent/CN113645969A/zh
Pending legal-status Critical Current

Links

Images

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)
CN202080025346.0A 2019-02-04 2020-02-03 用于治疗与溶酶体贮积症相关的症状和病症的方法 Pending CN113645969A (zh)

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 (1)

Publication Number Publication Date
CN113645969A true CN113645969A (zh) 2021-11-12

Family

ID=69743942

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202080025346.0A Pending CN113645969A (zh) 2019-02-04 2020-02-03 用于治疗与溶酶体贮积症相关的症状和病症的方法
CN202080025266.5A Pending CN113710249A (zh) 2019-02-04 2020-02-03 用于治疗溶酶体贮积症相关的症状和病症的方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202080025266.5A Pending CN113710249A (zh) 2019-02-04 2020-02-03 用于治疗溶酶体贮积症相关的症状和病症的方法

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
CN103917094A (zh) * 2011-03-18 2014-07-09 建新公司 葡萄糖基神经酰胺合酶抑制剂
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103917094A (zh) * 2011-03-18 2014-07-09 建新公司 葡萄糖基神经酰胺合酶抑制剂
US20180036295A1 (en) * 2015-03-10 2018-02-08 Genzyme Corporation Methods for treating proteinopathies
CN107872976A (zh) * 2015-03-10 2018-04-03 建新公司 用于治疗蛋白质病的方法

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ETTORE SALSANO等: "Vertical supranuclear gaze palsy in Niemann-Pick type C disease", vol. 33, no. 6, pages 1225 - 1232, XP035145354, DOI: 10.1007/s10072-012-1155-1 *
IDA BERGLIN ENQUIST等: ""Murine models of acute neuronopathic Gaucher disease"", 《PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA》, vol. 104, no. 44, 22 October 2007 (2007-10-22), pages 17483 - 17488 *
IDA BERGLIN ENQUIST等: "Murine models of acute neuronopathic Gaucher disease", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 104, no. 44, pages 17483 - 17488 *
S. PABLO SARDI等: ""Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models"", 《PROCEEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES》, vol. 114, no. 10, 21 February 2017 (2017-02-21), pages 2699 - 2704 *

Also Published As

Publication number Publication date
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
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

Similar Documents

Publication Publication Date Title
CN113645969A (zh) 用于治疗与溶酶体贮积症相关的症状和病症的方法
CN115427038A (zh) 治疗与溶酶体贮积病相关的神经系统症状的方法
CN107080747A (zh) 葡萄糖基神经酰胺合酶抑制剂
CN107872976A (zh) 用于治疗蛋白质病的方法
CA3187086A1 (en) Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
RU2824599C2 (ru) Способы лечения симптомов и нарушений, ассоциированных с лизосомными болезнями накопления
RU2829786C2 (ru) Способы лечения симптомов и нарушений, ассоциированных с лизосомными болезнями накопления
CA3128041C (en) Methods for treating symptoms and disorders associated with lysosomal storage diseases
RU2839414C1 (ru) Способы лечения неврологических симптомов, ассоциированных с лизосомными болезнями накопления

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination