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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Withdrawn
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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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Application filed by 젠자임 코포레이션 filed Critical 젠자임 코포레이션
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Withdrawn legal-status Critical Current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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (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 (enExample)
EP (2) EP3267983B1 (enExample)
JP (2) JP6990110B2 (enExample)
KR (1) KR20170123329A (enExample)
CN (1) CN107872976A (enExample)
AU (1) AU2016229826A1 (enExample)
CA (1) CA2978883A1 (enExample)
EA (1) EA201791993A1 (enExample)
ES (1) ES2973101T3 (enExample)
HK (1) HK1244217A1 (enExample)
HU (1) HUE065628T2 (enExample)
IL (1) IL254393A0 (enExample)
MX (1) MX2017011598A (enExample)
PL (1) PL3267983T3 (enExample)
PT (1) PT3267983T (enExample)
TW (1) TW201642855A (enExample)
WO (1) WO2016145046A1 (enExample)

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
BR112019027029A2 (pt) * 2017-06-19 2020-07-14 Kainos Medicine, Inc. moduladores de alfa-sinucleina
TWI644673B (zh) * 2017-07-27 2018-12-21 百朗克股份有限公司 頭孢曲松的用途
WO2020163244A1 (en) * 2019-02-04 2020-08-13 Genzyme Corporation Methods for treating symptoms and disorders associated with lysosomal storage diseases
US12527776B2 (en) 2019-02-04 2026-01-20 Genzyme Corporation Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS)
BR112022009409A2 (pt) * 2019-11-15 2022-08-09 Yuhan Corp Novos derivados que têm grupamento 2,3-dihidro-1h-indeno ou 2,3-dihidrobenzofurano ou sal farmaceuticamente aceitável dos mesmos e composições farmacêuticas que os compreendem
EP4100009A1 (en) 2020-02-03 2022-12-14 Genzyme Corporation Methods for treating neurological symptoms associated with lysosomal storage diseases
KR20230005813A (ko) 2020-02-28 2023-01-10 프락시스 프리시젼 메디신즈, 인크. Kcnt1 억제제 및 사용 방법
BR112022017530A2 (pt) 2020-03-17 2022-10-18 Sumitomo Pharma Co Ltd Derivado de oxadiazol
BR112022019041A2 (pt) * 2020-03-23 2022-12-27 Praxis Prec Medicines Inc Inibidores de kcnt1 e métodos de uso
KR20230043024A (ko) * 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
MX2023001285A (es) 2020-07-30 2023-02-22 Genzyme Corp Metodos para reducir la concentracion de glicoesfingolipidos en el tejido cerebral y metodos de tratamiento de enfermedades neurodegenerativas que implican los mismos.
US11773088B2 (en) 2020-11-02 2023-10-03 Praxis Precision Medicines, Inc. KCNT1 inhibitors and methods of use
KR20250106321A (ko) 2022-12-01 2025-07-09 젠자임 코포레이션 강력하거나 중간 수준인 cyp3a4 억제제와 조합한 벤글루스타트
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 (enExample) 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
CN103917094B (zh) * 2011-03-18 2016-11-09 建新公司 葡萄糖基神经酰胺合酶抑制剂
US9845327B2 (en) * 2011-06-22 2017-12-19 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
EP4349408A2 (en) 2024-04-10
ES2973101T3 (es) 2024-06-18
PL3267983T3 (pl) 2024-05-13
JP6990110B2 (ja) 2022-02-10
TW201642855A (zh) 2016-12-16
JP2021119185A (ja) 2021-08-12
IL254393A0 (en) 2017-11-30
US20180036295A1 (en) 2018-02-08
HUE065628T2 (hu) 2024-06-28
CA2978883A1 (en) 2016-09-15
JP2018507886A (ja) 2018-03-22
EA201791993A1 (ru) 2017-12-29
WO2016145046A1 (en) 2016-09-15
EP4349408A3 (en) 2024-06-19
EP3267983B1 (en) 2023-12-20
PT3267983T (pt) 2024-03-12
AU2016229826A1 (en) 2017-10-26
MX2017011598A (es) 2017-12-20
US20200197374A1 (en) 2020-06-25
JP7374149B2 (ja) 2023-11-06
HK1244217A1 (zh) 2018-08-03
EP3267983A1 (en) 2018-01-17
CN107872976A (zh) 2018-04-03

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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