IL215583A - Use of Medical Material 5- (Fluorine and Methyl) Pipridine-3,4-Diol, 5- (Chloromethyl) Pipridine-3,4-Diol for the preparation of a drug for the prevention and / or treatment of lysosomal storage disorders in patients at risk of disease or being diagnosed with these disorders - Google Patents
Use of Medical Material 5- (Fluorine and Methyl) Pipridine-3,4-Diol, 5- (Chloromethyl) Pipridine-3,4-Diol for the preparation of a drug for the prevention and / or treatment of lysosomal storage disorders in patients at risk of disease or being diagnosed with these disordersInfo
- Publication number
- IL215583A IL215583A IL215583A IL21558311A IL215583A IL 215583 A IL215583 A IL 215583A IL 215583 A IL215583 A IL 215583A IL 21558311 A IL21558311 A IL 21558311A IL 215583 A IL215583 A IL 215583A
- Authority
- IL
- Israel
- Prior art keywords
- piperidine
- diol
- theraprutic
- fluoromethyl
- chloromethyl
- Prior art date
Links
- KTXBXDGOZMHNIC-UHFFFAOYSA-N 5-(chloromethyl)piperidine-3,4-diol Chemical compound OC1CNCC(CCl)C1O KTXBXDGOZMHNIC-UHFFFAOYSA-N 0.000 title 1
- BEQPGACEJJJLEA-UHFFFAOYSA-N 5-(fluoromethyl)piperidine-3,4-diol Chemical compound OC1CNCC(CF)C1O BEQPGACEJJJLEA-UHFFFAOYSA-N 0.000 title 1
- 208000015439 Lysosomal storage disease Diseases 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 title 1
- 239000003814 drug Substances 0.000 title 1
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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16810909P | 2009-04-09 | 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 |
|---|---|
| IL215583A0 IL215583A0 (en) | 2011-12-29 |
| IL215583A true IL215583A (en) | 2016-09-29 |
Family
ID=42934559
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL215583A IL215583A (en) | 2009-04-09 | 2011-10-06 | Use of Medical Material 5- (Fluorine and Methyl) Pipridine-3,4-Diol, 5- (Chloromethyl) Pipridine-3,4-Diol for the preparation of a drug for the prevention and / or treatment of lysosomal storage disorders in patients at risk of disease or being diagnosed with these disorders |
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 |
|---|---|---|---|---|
| EP3782655A1 (en) | 2005-05-17 | 2021-02-24 | Amicus Therapeutics, Inc. | A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives |
| CA2758187C (en) * | 2009-04-09 | 2017-03-07 | Robert Boyd | Methods for preventing and/or treating degenerative disorders of the central nervous system |
| RU2015147509A (ru) | 2009-10-19 | 2019-01-14 | Амикус Терапьютикс, Инк. | Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы |
| WO2011049737A1 (en) | 2009-10-19 | 2011-04-28 | Amicus Therapeutics, Inc. | Novel compositions for preventing and/or treating lysosomal storage disorders |
| KR20200032244A (ko) | 2012-03-07 | 2020-03-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| CA2939380A1 (en) * | 2014-02-12 | 2015-08-20 | 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 |
| JP6851964B2 (ja) | 2014-09-30 | 2021-03-31 | アミカス セラピューティックス インコーポレイテッド | 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ |
| IL299470B2 (en) | 2015-12-30 | 2026-05-01 | Amicus Therapeutics Inc | Improved acid alpha-glucosidase for the treatment of Pompe disease |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| KR102618519B1 (ko) | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| CN119113091A (zh) | 2017-05-15 | 2024-12-13 | 阿米库斯治疗学公司 | 重组人类酸性α-葡萄糖苷酶 |
| 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 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4407957A (en) | 1981-03-13 | 1983-10-04 | Damon Corporation | Reversible microencapsulation of a core material |
| SG47470A1 (en) | 1991-04-25 | 1998-04-17 | Univ Brown Res Found | Implantable biocompatible immunoisolatory vehicle for delivery of a 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 |
| DK0679088T3 (da) | 1992-09-29 | 2002-10-28 | Inhale Therapeutic Syst | Pulmonal levering af aktive fragmenter af parathyreoideaea hormon |
| AU1890295A (en) * | 1994-03-09 | 1995-09-25 | Novo Nordisk A/S | Piperidines and pyrrolidines |
| 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 |
| WO2002076440A2 (en) * | 2001-03-23 | 2002-10-03 | Elan Pharmaceuticals, Inc. | Methods of treating alzheimer's disease with piperidin derivates |
| JP2006507359A (ja) | 2002-10-28 | 2006-03-02 | ノボ・ノルデイスク・エー／エス | 心循環器疾患の治療のためのグリコーゲンホスホリラーゼ阻害剤の使用 |
| US7446098B2 (en) | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US20050137223A1 (en) * | 2003-11-12 | 2005-06-23 | Amicus Therapeutics, Inc. | Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease |
| DK2040548T3 (da) * | 2006-06-23 | 2012-08-13 | Amicus Therapeutics Inc | Fremgangsmåde til behandling af neurologiske forstyrrelser ved forøgelse af aktiviteten af beta-glucocerebrosidase |
| AU2008245578A1 (en) | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| US8321148B2 (en) | 2008-10-24 | 2012-11-27 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| CA2758187C (en) * | 2009-04-09 | 2017-03-07 | Robert Boyd | Methods for preventing and/or treating degenerative disorders of the central nervous system |
-
2010
- 2010-04-09 JP JP2012504881A patent/JP5645918B2/ja not_active Expired - Fee Related
- 2010-04-09 US US12/757,160 patent/US8940766B2/en active Active
- 2010-04-09 CN CN201080023544.XA patent/CN102625661B/zh not_active Expired - Fee Related
- 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 BR BRPI1010517A patent/BRPI1010517A2/pt not_active Application Discontinuation
- 2010-04-09 AU AU2010233188A patent/AU2010233188B2/en not_active Ceased
- 2010-04-09 ES ES10762469.4T patent/ES2564093T3/es active Active
- 2010-04-09 WO PCT/US2010/030472 patent/WO2010118283A1/en not_active Ceased
- 2010-04-09 NZ NZ595629A patent/NZ595629A/xx not_active IP Right Cessation
-
2011
- 2011-10-06 IL IL215583A patent/IL215583A/en active IP Right Grant
Also Published As
| Publication number | Publication date |
|---|---|
| CA2758271C (en) | 2018-06-05 |
| EP2416656A4 (en) | 2012-09-05 |
| EP2416656B1 (en) | 2015-12-23 |
| CA2758271A1 (en) | 2010-10-14 |
| ES2564093T3 (es) | 2016-03-17 |
| NZ595629A (en) | 2013-04-26 |
| US8940766B2 (en) | 2015-01-27 |
| JP5645918B2 (ja) | 2014-12-24 |
| US20100260740A1 (en) | 2010-10-14 |
| WO2010118283A1 (en) | 2010-10-14 |
| BRPI1010517A2 (pt) | 2017-01-31 |
| IL215583A0 (en) | 2011-12-29 |
| EP2416656A1 (en) | 2012-02-15 |
| CN102625661A (zh) | 2012-08-01 |
| AU2010233188A1 (en) | 2011-11-03 |
| JP2012523431A (ja) | 2012-10-04 |
| CN102625661B (zh) | 2015-05-27 |
| AU2010233188B2 (en) | 2015-07-16 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FF | Patent granted | ||
| KB | Patent renewed | ||
| KB | Patent renewed |