JP2017502961A - グルコセレブロシダーゼモジュレーターおよびその使用 - Google Patents

グルコセレブロシダーゼモジュレーターおよびその使用 Download PDF

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Publication number
JP2017502961A
JP2017502961A JP2016543197A JP2016543197A JP2017502961A JP 2017502961 A JP2017502961 A JP 2017502961A JP 2016543197 A JP2016543197 A JP 2016543197A JP 2016543197 A JP2016543197 A JP 2016543197A JP 2017502961 A JP2017502961 A JP 2017502961A
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gcase
azaspiro
octane
diol
disease
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JP2017502961A5 (https=
Inventor
ラメシュ カウル
ラメシュ カウル
アーネスト ジェイ マッキーチャン
アーネスト ジェイ マッキーチャン
ジャンユ スン
ジャンユ スン
デイヴィッド ジェイ ヴォカドロ
デイヴィッド ジェイ ヴォカドロ
ユアンシー ジョウ
ユアンシー ジョウ
ヨンバオ ジュー
ヨンバオ ジュー
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アレクトス セラピューティックス インコーポレイテッド
アレクトス セラピューティックス インコーポレイテッド
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Publication of JP2017502961A publication Critical patent/JP2017502961A/ja
Publication of JP2017502961A5 publication Critical patent/JP2017502961A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D221/00—Heterocyclic compounds containing six-membered rings having one nitrogen atom as the only ring hetero atom, not provided for by groups C07D211/00 - C07D219/00
    • C07D221/02—Heterocyclic compounds containing six-membered rings having one nitrogen atom as the only ring hetero atom, not provided for by groups C07D211/00 - C07D219/00 condensed with carbocyclic rings or ring systems
    • C07D221/20—Spiro-condensed ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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

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  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Psychiatry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Other In-Based Heterocyclic Compounds (AREA)
JP2016543197A 2013-12-23 2014-12-22 グルコセレブロシダーゼモジュレーターおよびその使用 Pending JP2017502961A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361920160P 2013-12-23 2013-12-23
US61/920,160 2013-12-23
US201462005196P 2014-05-30 2014-05-30
US62/005,196 2014-05-30
PCT/CA2014/051252 WO2015095963A1 (en) 2013-12-23 2014-12-22 Glucocerebrosidase modulators and uses thereof

Publications (2)

Publication Number Publication Date
JP2017502961A true JP2017502961A (ja) 2017-01-26
JP2017502961A5 JP2017502961A5 (https=) 2018-02-08

Family

ID=53477250

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JP2016543197A Pending JP2017502961A (ja) 2013-12-23 2014-12-22 グルコセレブロシダーゼモジュレーターおよびその使用

Country Status (4)

Country Link
US (2) US9796680B2 (https=)
EP (1) EP3087056A4 (https=)
JP (1) JP2017502961A (https=)
WO (1) WO2015095963A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2022530017A (ja) * 2019-04-25 2022-06-27 武田薬品工業株式会社 イソファゴミン塩、その使用方法および製剤

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105631432B (zh) * 2016-01-04 2020-12-08 京东方科技集团股份有限公司 指纹探测电路及驱动方法和显示装置
EP3538095A4 (en) * 2016-11-14 2020-06-17 Berg LLC Methods for treating parkinson's disease
CN108530375B (zh) * 2018-05-29 2020-04-21 南京药石科技股份有限公司 4-氧杂-7-氮杂螺[2.5]辛烷或其盐的中间体及制备方法
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
US12331022B2 (en) 2019-05-02 2025-06-17 Merck Sharp & Dohme Llc Spiropiperidine allosteric modulators of nicotinic acetylcholine receptors
EP4329880A4 (en) * 2021-04-30 2025-03-05 Vanqua Bio, Inc. SMALL MOLECULE MODULATORS OF GLUCOCEREBROSIDASE ACTIVITY AND USES THEREOF
EP4329881A4 (en) * 2021-04-30 2025-06-11 Vanqua Bio, Inc. Small molecule modulators of glucocerebrosidase activity and uses thereof
KR20240102969A (ko) * 2021-10-13 2024-07-03 밴쿠아 바이오, 인크. 글루코세레브로시다제 활성의 소분자 조절제 및 이의 용도

Citations (5)

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JP2007512253A (ja) * 2003-11-12 2007-05-17 アミカス セラピューティックス インコーポレイテッド ゴーシェ病を治療するための、ヒドロキシピペリジン誘導体
JP2008523145A (ja) * 2004-12-13 2008-07-03 イーライ リリー アンド カンパニー リポキシゲナーゼ阻害剤としてのスピロ誘導体
WO2012058193A1 (en) * 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. Leucine-rich repeat kinase enzyme activity
JP2013508366A (ja) * 2009-10-19 2013-03-07 アミカス セラピューティックス インコーポレイテッド 中枢神経系の変性障害を予防および／または治療するための新規の組成物
WO2013075227A1 (en) * 2011-11-25 2013-05-30 The Hospital For Sick Children β-GLUCOCEREBROSIDASE CHAPERONES

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JP5303458B2 (ja) 2006-06-23 2013-10-02 アミークス セラピューティックス インコーポレイテッド β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
CA2758187C (en) 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
CA2868459A1 (en) 2012-03-27 2013-10-03 Amicus Therapeutics, Inc. Polymorphs of (3r,4r,5s)-5-(difluoromethyl)piperidine-3,4-diol fumarate and uses thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007512253A (ja) * 2003-11-12 2007-05-17 アミカス セラピューティックス インコーポレイテッド ゴーシェ病を治療するための、ヒドロキシピペリジン誘導体
JP2008523145A (ja) * 2004-12-13 2008-07-03 イーライ リリー アンド カンパニー リポキシゲナーゼ阻害剤としてのスピロ誘導体
JP2013508366A (ja) * 2009-10-19 2013-03-07 アミカス セラピューティックス インコーポレイテッド 中枢神経系の変性障害を予防および／または治療するための新規の組成物
WO2012058193A1 (en) * 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. Leucine-rich repeat kinase enzyme activity
WO2013075227A1 (en) * 2011-11-25 2013-05-30 The Hospital For Sick Children β-GLUCOCEREBROSIDASE CHAPERONES

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PEARSON, M. S. M. ET AL.: "The spirocyclopropyl moiety as a methyl surrogate in the structure of L-fucosidase and L-rhamnosidas", BIOORGANIC & MEDICINAL CHEMISTRY, vol. 17(23), JPN6018039896, 2009, pages 8020 - 8026, ISSN: 0003896053 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2022530017A (ja) * 2019-04-25 2022-06-27 武田薬品工業株式会社 イソファゴミン塩、その使用方法および製剤
JP7642561B2 (ja) 2019-04-25 2025-03-10 武田薬品工業株式会社 イソファゴミン塩、その使用方法および製剤
JP2025084834A (ja) * 2019-04-25 2025-06-03 武田薬品工業株式会社 イソファゴミン塩、その使用方法および製剤

Also Published As

Publication number Publication date
US10081601B2 (en) 2018-09-25
US20180009761A1 (en) 2018-01-11
EP3087056A1 (en) 2016-11-02
US9796680B2 (en) 2017-10-24
WO2015095963A1 (en) 2015-07-02
EP3087056A4 (en) 2017-07-19
US20170029379A1 (en) 2017-02-02

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