CN107872976A - 用于治疗蛋白质病的方法 - Google Patents

用于治疗蛋白质病的方法 Download PDF

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Publication number
CN107872976A
CN107872976A CN201680026839.XA CN201680026839A CN107872976A CN 107872976 A CN107872976 A CN 107872976A CN 201680026839 A CN201680026839 A CN 201680026839A CN 107872976 A CN107872976 A CN 107872976A
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CN
China
Prior art keywords
disease
compound
subject
protein
prodrug
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
CN201680026839.XA
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English (en)
Chinese (zh)
Inventor
S·H·程
L·施哈布丁
S·P·萨尔迪
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 CN107872976A publication Critical patent/CN107872976A/zh
Pending legal-status Critical Current

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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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D453/00—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02—Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
CN201680026839.XA 2015-03-10 2016-03-09 用于治疗蛋白质病的方法 Pending CN107872976A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562131071P 2015-03-10 2015-03-10
US62/131,071 2015-03-10
PCT/US2016/021512 WO2016145046A1 (en) 2015-03-10 2016-03-09 Methods for treating proteinopathies

Publications (1)

Publication Number Publication Date
CN107872976A true CN107872976A (zh) 2018-04-03

Family

ID=55587378

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680026839.XA Pending CN107872976A (zh) 2015-03-10 2016-03-09 用于治疗蛋白质病的方法

Country Status (17)

Country Link
US (2) US20180036295A1 (https=)
EP (2) EP3267983B1 (https=)
JP (2) JP6990110B2 (https=)
KR (1) KR20170123329A (https=)
CN (1) CN107872976A (https=)
AU (1) AU2016229826A1 (https=)
CA (1) CA2978883A1 (https=)
EA (1) EA201791993A1 (https=)
ES (1) ES2973101T3 (https=)
HK (1) HK1244217A1 (https=)
HU (1) HUE065628T2 (https=)
IL (1) IL254393A0 (https=)
MX (1) MX2017011598A (https=)
PL (1) PL3267983T3 (https=)
PT (1) PT3267983T (https=)
TW (1) TW201642855A (https=)
WO (1) WO2016145046A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113645969A (zh) * 2019-02-04 2021-11-12 建新公司 用于治疗与溶酶体贮积症相关的症状和病症的方法
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
CN116133647A (zh) * 2020-07-24 2023-05-16 建新公司 包含文鲁司他的药物组合物

Families Citing this family (12)

* 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
WO2018234864A2 (en) 2017-06-19 2018-12-27 Kainos Medicine Inc. ALPHA-SYNUCLEINE MODULATORS
TWI644673B (zh) * 2017-07-27 2018-12-21 百朗克股份有限公司 頭孢曲松的用途
PL4041733T3 (pl) * 2019-11-15 2024-09-23 Yuhan Corporation Pochodne mające ugrupowanie 2,3-dihydro-1h-indenu lub 2,3-dihydrobenzofuranu lub ich farmaceutycznie dopuszczalne sole oraz kompozycje farmaceutyczne je zawierające
TW202142236A (zh) 2020-02-03 2021-11-16 美商健臻公司 用於治療與溶體儲積症相關的神經症狀之方法
JP7699836B2 (ja) 2020-02-28 2025-06-30 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１阻害剤、および使用方法
DK4122923T3 (da) * 2020-03-17 2025-12-15 Sumitomo Pharma Co Ltd Oxadiazolderivat
KR20230005168A (ko) * 2020-03-23 2023-01-09 프락시스 프리시젼 메디신즈, 인크. Kcnt1 억제제 및 사용 방법
EP4188381A1 (en) * 2020-07-30 2023-06-07 Genzyme Corporation Methods for reducing glycosphingolipid concentration in brain tissue and methods of treatment of neurodegenerative diseases involving the same
US11773088B2 (en) 2020-11-02 2023-10-03 Praxis Precision Medicines, Inc. KCNT1 inhibitors and methods of use
CN120417900A (zh) 2022-12-01 2025-08-01 建新公司 文鲁司他与cyp3a4的强或中度抑制剂的组合
WO2025262570A1 (en) 2024-06-17 2025-12-26 Genzyme Corporation Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
WO2014043068A1 (en) * 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors
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

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US4683A (en) 1846-08-08 waring and richard e
US195A (en) 1837-05-15 Machine fob cutting and dressing stone
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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences

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
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
WO2014043068A1 (en) * 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Non-Patent Citations (2)

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Title
（英）博格斯拉夫斯基、（英）费舍尔主编: "《神经病学》", 30 April 2004, 天津科学技术出版社 *
蒋雨平等著: "《新编神经疾病学》", 31 October 2014, 上海科学普及出版社 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113645969A (zh) * 2019-02-04 2021-11-12 建新公司 用于治疗与溶酶体贮积症相关的症状和病症的方法
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
CN116133647A (zh) * 2020-07-24 2023-05-16 建新公司 包含文鲁司他的药物组合物

Also Published As

Publication number Publication date
PT3267983T (pt) 2024-03-12
EP3267983A1 (en) 2018-01-17
JP2018507886A (ja) 2018-03-22
JP2021119185A (ja) 2021-08-12
JP7374149B2 (ja) 2023-11-06
MX2017011598A (es) 2017-12-20
KR20170123329A (ko) 2017-11-07
PL3267983T3 (pl) 2024-05-13
HK1244217A1 (zh) 2018-08-03
AU2016229826A1 (en) 2017-10-26
TW201642855A (zh) 2016-12-16
EP4349408A2 (en) 2024-04-10
EA201791993A1 (ru) 2017-12-29
WO2016145046A1 (en) 2016-09-15
JP6990110B2 (ja) 2022-02-10
EP3267983B1 (en) 2023-12-20
HUE065628T2 (hu) 2024-06-28
ES2973101T3 (es) 2024-06-18
CA2978883A1 (en) 2016-09-15
US20180036295A1 (en) 2018-02-08
EP4349408A3 (en) 2024-06-19
IL254393A0 (en) 2017-11-30
US20200197374A1 (en) 2020-06-25

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Application publication date: 20180403