KR20170123329A - 단백질병증을 치료하기 위한 방법 - Google Patents

단백질병증을 치료하기 위한 방법 Download PDF

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Publication number
KR20170123329A
KR20170123329A KR1020177027604A KR20177027604A KR20170123329A KR 20170123329 A KR20170123329 A KR 20170123329A KR 1020177027604 A KR1020177027604 A KR 1020177027604A KR 20177027604 A KR20177027604 A KR 20177027604A KR 20170123329 A KR20170123329 A KR 20170123329A
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KR
South Korea
Prior art keywords
compound
disease
subject
pharmaceutically acceptable
prodrug
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KR1020177027604A
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English (en)
Korean (ko)
Inventor
셍 에이치. 쳉
람야 쉬하부딘
세르지오 파블로 샤르디
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젠자임 코포레이션
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Publication of KR20170123329A publication Critical patent/KR20170123329A/ko
Withdrawn legal-status Critical Current

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    • 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
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D453/00Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids
    • C07D453/02Heterocyclic compounds containing quinuclidine or iso-quinuclidine ring systems, e.g. quinine alkaloids containing not further condensed quinuclidine ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration

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  • 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)
KR1020177027604A 2015-03-10 2016-03-09 단백질병증을 치료하기 위한 방법 Withdrawn KR20170123329A (ko)

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
KR20170123329A true KR20170123329A (ko) 2017-11-07

Family

ID=55587378

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020177027604A Withdrawn KR20170123329A (ko) 2015-03-10 2016-03-09 단백질병증을 치료하기 위한 방법

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=)

Families Citing this family (15)

* 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
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
AU2020218496B2 (en) * 2019-02-04 2024-12-19 Genzyme Corporation Methods for treating symptoms and disorders associated with lysosomal storage diseases
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
KR20230043024A (ko) * 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2012231275A1 (en) * 2011-03-18 2013-10-17 Genzyme Corporation Glucosylceramide synthase inhibitors
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
MA37975B2 (fr) * 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide

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
PL3267983T3 (pl) 2024-05-13
HK1244217A1 (zh) 2018-08-03
AU2016229826A1 (en) 2017-10-26
CN107872976A (zh) 2018-04-03
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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Date Code Title Description
PA0105 International application

Patent event date: 20170928

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination