CN102625661B - 用于预防和/或治疗溶酶体贮积失调的方法 - Google Patents
用于预防和/或治疗溶酶体贮积失调的方法 Download PDFInfo
- Publication number
- CN102625661B CN102625661B CN201080023544.XA CN201080023544A CN102625661B CN 102625661 B CN102625661 B CN 102625661B CN 201080023544 A CN201080023544 A CN 201080023544A CN 102625661 B CN102625661 B CN 102625661B
- Authority
- CN
- China
- Prior art keywords
- piperidines
- glycol
- chloromethyl
- compound
- purposes
- 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
Links
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)
- Biotechnology (AREA)
- Biophysics (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Hydrogenated Pyridines (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 (2)
| Publication Number | Publication Date |
|---|---|
| CN102625661A CN102625661A (zh) | 2012-08-01 |
| CN102625661B true CN102625661B (zh) | 2015-05-27 |
Family
ID=42934559
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201080023544.XA Expired - Fee Related CN102625661B (zh) | 2009-04-09 | 2010-04-09 | 用于预防和/或治疗溶酶体贮积失调的方法 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US8940766B2 (enExample) |
| EP (1) | EP2416656B1 (enExample) |
| JP (1) | JP5645918B2 (enExample) |
| CN (1) | CN102625661B (enExample) |
| AU (1) | AU2010233188B2 (enExample) |
| BR (1) | BRPI1010517A2 (enExample) |
| CA (1) | CA2758271C (enExample) |
| ES (1) | ES2564093T3 (enExample) |
| IL (1) | IL215583A (enExample) |
| NZ (1) | NZ595629A (enExample) |
| WO (1) | WO2010118283A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2932982B1 (en) | 2005-05-17 | 2018-10-03 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| US8304429B2 (en) * | 2009-04-09 | 2012-11-06 | Amicus Therapeutics, Inc. | Methods for preventing and/or treating degenerative disorders of the central nervous system |
| EP2490533B1 (en) | 2009-10-19 | 2015-09-16 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
| RU2015147509A (ru) | 2009-10-19 | 2019-01-14 | Амикус Терапьютикс, Инк. | Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы |
| KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| MX2016010365A (es) * | 2014-02-12 | 2016-11-30 | Amicus Therapeutics Inc | Derivados de azucares que comprenden restos que contienen azufre, metodos para prepararlos y metodos para emplearlos en el tratamiento de mps iiic. |
| SI4273241T1 (sl) | 2014-09-30 | 2025-03-31 | Amicus Therapeutics, Inc. | Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati |
| KR102510941B1 (ko) | 2015-12-30 | 2023-03-20 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| NZ786723A (en) | 2016-03-30 | 2025-07-25 | Amicus Therapeutics Inc | Method for selection of high m6p recombinant proteins |
| CA3019128A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| DK3624831T5 (da) | 2017-05-15 | 2024-09-02 | Amicus Therapeutics Inc | Rekombinant human sur alfa-glucosidase |
| WO2020023390A1 (en) | 2018-07-25 | 2020-01-30 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040204379A1 (en) * | 2000-06-19 | 2004-10-14 | Cheng Seng H. | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US20050130972A1 (en) * | 2003-11-12 | 2005-06-16 | Amicus Therapeutics, Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
| US20060079533A1 (en) * | 2001-03-23 | 2006-04-13 | Nieman James A | Methods of treating alzheimer's disease |
| US20080009516A1 (en) * | 2006-06-23 | 2008-01-10 | Wustman Brandon A | Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4407957A (en) | 1981-03-13 | 1983-10-04 | Damon Corporation | Reversible microencapsulation of a core material |
| CA2109085C (en) | 1991-04-25 | 2003-03-11 | Keith E. Dionne | Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products |
| 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 |
| ES2179831T3 (es) | 1992-09-29 | 2003-02-01 | Inhale Therapeutic Syst | Liberacion en los pulmones de fragmentos activos de hormona paratiroidiana. |
| 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 |
| 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 |
| WO2008134628A2 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| US8304429B2 (en) * | 2009-04-09 | 2012-11-06 | Amicus Therapeutics, Inc. | Methods for preventing and/or treating degenerative disorders of the central nervous system |
-
2010
- 2010-04-09 CA CA2758271A patent/CA2758271C/en not_active Expired - Fee Related
- 2010-04-09 EP EP10762469.4A patent/EP2416656B1/en not_active Not-in-force
- 2010-04-09 CN CN201080023544.XA patent/CN102625661B/zh not_active Expired - Fee Related
- 2010-04-09 NZ NZ595629A patent/NZ595629A/xx not_active IP Right Cessation
- 2010-04-09 AU AU2010233188A patent/AU2010233188B2/en not_active Ceased
- 2010-04-09 BR BRPI1010517A patent/BRPI1010517A2/pt not_active Application Discontinuation
- 2010-04-09 US US12/757,160 patent/US8940766B2/en active Active
- 2010-04-09 ES ES10762469.4T patent/ES2564093T3/es active Active
- 2010-04-09 JP JP2012504881A patent/JP5645918B2/ja not_active Expired - Fee Related
- 2010-04-09 WO PCT/US2010/030472 patent/WO2010118283A1/en not_active Ceased
-
2011
- 2011-10-06 IL IL215583A patent/IL215583A/en active IP Right Grant
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20050130972A1 (en) * | 2003-11-12 | 2005-06-16 | Amicus Therapeutics, Inc. | Hydroxy piperidine derivatives to treat gaucher disease |
| US20080009516A1 (en) * | 2006-06-23 | 2008-01-10 | Wustman Brandon A | Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase |
Also Published As
| Publication number | Publication date |
|---|---|
| AU2010233188B2 (en) | 2015-07-16 |
| US8940766B2 (en) | 2015-01-27 |
| EP2416656A4 (en) | 2012-09-05 |
| WO2010118283A1 (en) | 2010-10-14 |
| BRPI1010517A2 (pt) | 2017-01-31 |
| EP2416656B1 (en) | 2015-12-23 |
| NZ595629A (en) | 2013-04-26 |
| IL215583A0 (en) | 2011-12-29 |
| JP2012523431A (ja) | 2012-10-04 |
| AU2010233188A1 (en) | 2011-11-03 |
| EP2416656A1 (en) | 2012-02-15 |
| JP5645918B2 (ja) | 2014-12-24 |
| CA2758271A1 (en) | 2010-10-14 |
| ES2564093T3 (es) | 2016-03-17 |
| US20100260740A1 (en) | 2010-10-14 |
| IL215583A (en) | 2016-09-29 |
| CA2758271C (en) | 2018-06-05 |
| CN102625661A (zh) | 2012-08-01 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CN102625661B (zh) | 用于预防和/或治疗溶酶体贮积失调的方法 | |
| TWI558396B (zh) | 用於預防和/或治療溶酶體貯積失調之新穎組合物 | |
| HK1174201B (en) | Novel compositions for preventing and/or treating lysosomal storage disorders |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| C06 | Publication | ||
| PB01 | Publication | ||
| C10 | Entry into substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| C14 | Grant of patent or utility model | ||
| GR01 | Patent grant | ||
| CF01 | Termination of patent right due to non-payment of annual fee |
Granted publication date: 20150527 Termination date: 20210409 |
|
| CF01 | Termination of patent right due to non-payment of annual fee |