JP5645918B2 - リソソーム蓄積障害の予防および／または治療方法 - Google Patents

リソソーム蓄積障害の予防および／または治療方法 Download PDF

Info

Publication number
JP5645918B2
JP5645918B2 JP2012504881A JP2012504881A JP5645918B2 JP 5645918 B2 JP5645918 B2 JP 5645918B2 JP 2012504881 A JP2012504881 A JP 2012504881A JP 2012504881 A JP2012504881 A JP 2012504881A JP 5645918 B2 JP5645918 B2 JP 5645918B2
Authority
JP
Japan
Prior art keywords
compound
diol
piperidine
administered
therapeutic agent
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.)
Expired - Fee Related
Application number
JP2012504881A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012523431A (ja
JP2012523431A5 (https=
Inventor
ロバート・ボイド
ゲイリー・リー
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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 アミカス セラピューティックス インコーポレイテッド, アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2012523431A publication Critical patent/JP2012523431A/ja
Publication of JP2012523431A5 publication Critical patent/JP2012523431A5/ja
Application granted granted Critical
Publication of JP5645918B2 publication Critical patent/JP5645918B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2012504881A 2009-04-09 2010-04-09 リソソーム蓄積障害の予防および／または治療方法 Expired - Fee Related JP5645918B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16810909P 2009-04-09 2009-04-09
US61/168,109 2009-04-09
PCT/US2010/030472 WO2010118283A1 (en) 2009-04-09 2010-04-09 Methods for preventing and/or treating lysosomal storage disorders

Publications (3)

Publication Number Publication Date
JP2012523431A JP2012523431A (ja) 2012-10-04
JP2012523431A5 JP2012523431A5 (https=) 2013-05-16
JP5645918B2 true JP5645918B2 (ja) 2014-12-24

Family

ID=42934559

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2012504881A Expired - Fee Related JP5645918B2 (ja) 2009-04-09 2010-04-09 リソソーム蓄積障害の予防および／または治療方法

Country Status (11)

Country Link
US (1) US8940766B2 (https=)
EP (1) EP2416656B1 (https=)
JP (1) JP5645918B2 (https=)
CN (1) CN102625661B (https=)
AU (1) AU2010233188B2 (https=)
BR (1) BRPI1010517A2 (https=)
CA (1) CA2758271C (https=)
ES (1) ES2564093T3 (https=)
IL (1) IL215583A (https=)
NZ (1) NZ595629A (https=)
WO (1) WO2010118283A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3782655A1 (en) 2005-05-17 2021-02-24 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
BRPI1015472A2 (pt) * 2009-04-09 2015-11-24 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças degenerativas do sistema nervoso central
KR101880411B1 (ko) 2009-10-19 2018-07-19 아미쿠스 세라퓨틱스, 인코포레이티드 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물
SI2995306T1 (sl) 2009-10-19 2019-04-30 Amicus Therapeutics, Inc. Novi sestavki za preprečevanje in/ali zdravljenje motenj lizosomskega shranjevanja
KR20230066482A (ko) * 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
US9598438B2 (en) * 2014-02-12 2017-03-21 Amicus Therapeutics, Inc. Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of MPS IIIC
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
KR20250007693A (ko) 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
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

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
ATE156344T1 (de) 1991-04-25 1997-08-15 Univ Brown Res Found Implantierbare, biokompatible immunisolator- trägersubstanz zum abgeben ausgesuchter, therapeutischer produkte
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
EP0679088B1 (en) 1992-09-29 2002-07-10 Inhale Therapeutic Systems Pulmonary delivery of active fragments of parathyroid hormone
ATE243196T1 (de) 1994-03-09 2003-07-15 Novo Nordisk As Piperidine und pyrrolidine
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5844102A (en) 1994-07-07 1998-12-01 University Of Maryland Baltimore County Glycohydrolase inhibitors, their preparation and use thereof
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20060079533A1 (en) * 2001-03-23 2006-04-13 Nieman James A Methods of treating alzheimer's disease
EP1558245A2 (en) 2002-10-28 2005-08-03 Novo Nordisk A/S Use of glycogen phosphorylase inhibitors for treatment of cardiovascular diseases
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
CA2545433A1 (en) * 2003-11-12 2005-05-26 Amicus Therapeutics Inc. Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease
WO2007150064A2 (en) * 2006-06-23 2007-12-27 Amicus Therapeutics, Inc. METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE
MX2009011473A (es) * 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
BRPI1015472A2 (pt) * 2009-04-09 2015-11-24 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças degenerativas do sistema nervoso central

Also Published As

Publication number Publication date
AU2010233188B2 (en) 2015-07-16
WO2010118283A1 (en) 2010-10-14
AU2010233188A1 (en) 2011-11-03
CA2758271C (en) 2018-06-05
JP2012523431A (ja) 2012-10-04
CN102625661B (zh) 2015-05-27
IL215583A (en) 2016-09-29
IL215583A0 (en) 2011-12-29
ES2564093T3 (es) 2016-03-17
US8940766B2 (en) 2015-01-27
CN102625661A (zh) 2012-08-01
EP2416656B1 (en) 2015-12-23
EP2416656A4 (en) 2012-09-05
NZ595629A (en) 2013-04-26
EP2416656A1 (en) 2012-02-15
BRPI1010517A2 (pt) 2017-01-31
US20100260740A1 (en) 2010-10-14
CA2758271A1 (en) 2010-10-14

Similar Documents

Publication Publication Date Title
JP5645918B2 (ja) リソソーム蓄積障害の予防および／または治療方法
JP6147005B2 (ja) リソソーム蓄積障害を予防および／または治療するための新規の組成物
JP5634498B2 (ja) 中枢神経系の変性障害の予防および／または治療方法
HK1174201B (en) Novel compositions for preventing and/or treating lysosomal storage disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130327

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20130327

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20131219

RD03 Notification of appointment of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7423

Effective date: 20140417

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140422

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20140502

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140718

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140728

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140822

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20141007

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20141104

R150 Certificate of patent or registration of utility model

Ref document number: 5645918

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees