JP6990110B2 - タンパク質症を処置するための方法 - Google Patents

タンパク質症を処置するための方法 Download PDF

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Publication number
JP6990110B2
JP6990110B2 JP2017547448A JP2017547448A JP6990110B2 JP 6990110 B2 JP6990110 B2 JP 6990110B2 JP 2017547448 A JP2017547448 A JP 2017547448A JP 2017547448 A JP2017547448 A JP 2017547448A JP 6990110 B2 JP6990110 B2 JP 6990110B2
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compound
quinuclidine
mice
disease
proteinosis
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JP2017547448A
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English (en)
Japanese (ja)
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JP2018507886A5 (enExample
JP2018507886A (ja
Inventor
エイチ・セン・チェン
ラムヤ・シハブディン
パブロ・セルジオ・サルディ
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Genzyme Corp
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Genzyme Corp
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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

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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)
JP2017547448A 2015-03-10 2016-03-09 タンパク質症を処置するための方法 Active JP6990110B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021078459A JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

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

Related Child Applications (1)

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JP2021078459A Division JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

Publications (3)

Publication Number Publication Date
JP2018507886A JP2018507886A (ja) 2018-03-22
JP2018507886A5 JP2018507886A5 (enExample) 2019-04-25
JP6990110B2 true JP6990110B2 (ja) 2022-02-10

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JP2017547448A Active JP6990110B2 (ja) 2015-03-10 2016-03-09 タンパク質症を処置するための方法
JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

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JP2021078459A Active JP7374149B2 (ja) 2015-03-10 2021-05-06 タンパク質症を処置するための方法

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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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Family Cites Families (10)

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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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014043068A1 (en) 2012-09-11 2014-03-20 Genzyme Corporation Glucosylceramide synthase inhibitors

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
J Neurochem，2014年，129(5)，884-894

Also Published As

Publication number Publication date
EP4349408A2 (en) 2024-04-10
ES2973101T3 (es) 2024-06-18
PL3267983T3 (pl) 2024-05-13
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
KR20170123329A (ko) 2017-11-07
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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