JP2002531505A - 糖脂質蓄積症を治療する薬物製造のためのデオキシノジリマイシンの長鎖ｎ−アルキル誘導体の使用 - Google Patents
糖脂質蓄積症を治療する薬物製造のためのデオキシノジリマイシンの長鎖ｎ−アルキル誘導体の使用Info
- Publication number
- JP2002531505A JP2002531505A JP2000586335A JP2000586335A JP2002531505A JP 2002531505 A JP2002531505 A JP 2002531505A JP 2000586335 A JP2000586335 A JP 2000586335A JP 2000586335 A JP2000586335 A JP 2000586335A JP 2002531505 A JP2002531505 A JP 2002531505A
- Authority
- JP
- Japan
- Prior art keywords
- dnj
- nonyl
- chain
- deoxynojirimycin
- alkyl
- 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.)
- Pending
Links
- LXBIFEVIBLOUGU-UHFFFAOYSA-N Deoxymannojirimycin Natural products OCC1NCC(O)C(O)C1O LXBIFEVIBLOUGU-UHFFFAOYSA-N 0.000 title claims abstract description 30
- 229930186217 Glycolipid Natural products 0.000 title claims abstract description 30
- HVCOBJNICQPDBP-UHFFFAOYSA-N 3-[3-[3,5-dihydroxy-6-methyl-4-(3,4,5-trihydroxy-6-methyloxan-2-yl)oxyoxan-2-yl]oxydecanoyloxy]decanoic acid;hydrate Chemical compound O.OC1C(OC(CC(=O)OC(CCCCCCC)CC(O)=O)CCCCCCC)OC(C)C(O)C1OC1C(O)C(O)C(O)C(C)O1 HVCOBJNICQPDBP-UHFFFAOYSA-N 0.000 title claims abstract description 27
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- 125000000217 alkyl group Chemical group 0.000 claims abstract description 13
- 125000004432 carbon atom Chemical group C* 0.000 claims abstract description 5
- FTSCEGKYKXESFF-LXTVHRRPSA-N N-nonyldeoxynojirimycin Chemical compound CCCCCCCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO FTSCEGKYKXESFF-LXTVHRRPSA-N 0.000 claims description 33
- 239000003085 diluting agent Substances 0.000 claims description 8
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- 125000001400 nonyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 abstract description 3
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- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 description 9
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- HSCJRCZFDFQWRP-UHFFFAOYSA-N Uridindiphosphoglukose Natural products OC1C(O)C(O)C(CO)OC1OP(O)(=O)OP(O)(=O)OCC1C(O)C(O)C(N2C(NC(=O)C=C2)=O)O1 HSCJRCZFDFQWRP-UHFFFAOYSA-N 0.000 description 2
- 239000013543 active substance Substances 0.000 description 2
- WPIHMWBQRSAMDE-YCZTVTEBSA-N beta-D-galactosyl-(1->4)-beta-D-galactosyl-N-(pentacosanoyl)sphingosine Chemical compound CCCCCCCCCCCCCCCCCCCCCCCCC(=O)N[C@@H](CO[C@@H]1O[C@H](CO)[C@H](O[C@@H]2O[C@H](CO)[C@H](O)[C@H](O)[C@H]2O)[C@H](O)[C@H]1O)[C@H](O)\C=C\CCCCCCCCCCCCC WPIHMWBQRSAMDE-YCZTVTEBSA-N 0.000 description 2
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- 208000024720 Fabry Disease Diseases 0.000 description 1
- PNNNRSAQSRJVSB-SLPGGIOYSA-N Fucose Natural products C[C@H](O)[C@@H](O)[C@H](O)[C@H](O)C=O PNNNRSAQSRJVSB-SLPGGIOYSA-N 0.000 description 1
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- 229940127551 Glucosyl Transferase Inhibitors Drugs 0.000 description 1
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- 125000001204 arachidyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 description 1
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- 229910052757 nitrogen Inorganic materials 0.000 description 1
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- QIQXTHQIDYTFRH-UHFFFAOYSA-N octadecanoic acid Chemical compound CCCCCCCCCCCCCCCCCC(O)=O QIQXTHQIDYTFRH-UHFFFAOYSA-N 0.000 description 1
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- 125000000913 palmityl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 description 1
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Classifications
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Hydrogenated Pyridines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11168398P | 1998-12-10 | 1998-12-10 | |
| US60/111,683 | 1998-12-10 | ||
| PCT/US1999/027918 WO2000033843A1 (en) | 1998-12-10 | 1999-12-08 | Use of long-chain n-alkyl derivatives of deoxynojirimycin for the manufacture of a medicament for the treatment of glycolipid storage diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2002531505A true JP2002531505A (ja) | 2002-09-24 |
| JP2002531505A5 JP2002531505A5 (https=) | 2007-01-11 |
Family
ID=22339901
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2000586335A Pending JP2002531505A (ja) | 1998-12-10 | 1999-12-08 | 糖脂質蓄積症を治療する薬物製造のためのデオキシノジリマイシンの長鎖ｎ−アルキル誘導体の使用 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US6610703B1 (https=) |
| EP (1) | EP1137416B1 (https=) |
| JP (1) | JP2002531505A (https=) |
| AT (1) | ATE226821T1 (https=) |
| AU (1) | AU2347500A (https=) |
| CA (1) | CA2352544C (https=) |
| DE (1) | DE69903750T2 (https=) |
| DK (1) | DK1137416T3 (https=) |
| ES (1) | ES2190674T3 (https=) |
| PT (1) | PT1137416E (https=) |
| WO (1) | WO2000033843A1 (https=) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6465488B1 (en) | 1997-12-11 | 2002-10-15 | Chancellor, Masters & Scholars Of The University Of Oxford | Inhibition of glycolipid biosynthesis |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| GB9909064D0 (en) * | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
| GB0100889D0 (en) | 2001-01-12 | 2001-02-21 | Oxford Glycosciences Uk Ltd | Compounds |
| GB9909066D0 (en) * | 1999-04-20 | 1999-06-16 | Oxford Glycosciences Uk Ltd | Therapies |
| AU2001269923A1 (en) * | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7994198B2 (en) | 2002-07-17 | 2011-08-09 | Actelion Pharmaceuticals Ltd. | Piperidinetriol derivatives as inhibitors of glycosylceramidsynthase |
| AU2003246942B2 (en) | 2002-07-17 | 2009-06-25 | Idorsia Pharmaceuticals Ltd | Piperidinetriol derivatives as inhibitors of glycosylceramide synthase |
| GB0313678D0 (en) | 2003-06-13 | 2003-07-16 | Oxford Glycosciences Uk Ltd | Novel compounds |
| GB0313677D0 (en) | 2003-06-13 | 2003-07-16 | Oxford Glycosciences Uk Ltd | Novel compound |
| EP1811991B1 (en) * | 2004-11-10 | 2018-11-07 | Genzyme Corporation | Treatment of type 2 diabetes using inhibitors of glycosphingolipid synthesis |
| SI2032134T1 (sl) | 2006-05-09 | 2015-10-30 | Genzyme Corporation | Postopki zdravljenja bolezni zamaščenih jeter, ki obsegajo inhibicijo sintezo glukosfingolipida |
| GB0614947D0 (en) * | 2006-07-27 | 2006-09-06 | Isis Innovation | Epitope reduction therapy |
| US8097728B2 (en) * | 2007-04-30 | 2012-01-17 | Philadelphia Health & Education Corporation | Iminosugar compounds with antiflavirus activity |
| EP2594564B1 (en) | 2007-05-31 | 2016-09-28 | Genzyme Corporation | 2-acylaminopropanol-type glucosylceramide synthase inhibitors |
| CN101888840A (zh) | 2007-10-05 | 2010-11-17 | 简詹姆公司 | 使用脑酰胺衍生物治疗多囊性肾疾病的方法 |
| CA2731685A1 (en) | 2008-07-28 | 2010-02-04 | Genzyme Corporation | Glucosylceramide synthase inhibition for the treatment of collapsing glomerulopathy and other glomerular disease |
| EP3078373A1 (en) | 2008-10-03 | 2016-10-12 | Genzyme Corporation | 2-acylaminopropanol-type glucosylceramide synthase inhibitor |
| CN102369023A (zh) | 2008-12-16 | 2012-03-07 | 建新公司 | 寡糖-蛋白缀合物 |
| EP2398321B1 (en) | 2009-02-23 | 2015-11-25 | United Therapeutics Corporation | Iminosugars and methods of treating viral diseases |
| EP2400843B1 (en) * | 2009-02-24 | 2015-11-11 | United Therapeutics Corporation | Iminosugars and methods of treating arenaviral infections |
| EP2411526A4 (en) * | 2009-03-27 | 2012-09-19 | Zacharon Pharmaceuticals Inc | MODULATORS OF GANGLIOSID BIOSYNTHESIS |
| ES2524361T3 (es) * | 2009-06-12 | 2014-12-05 | United Therapeutics Corporation | Iminoazúcares para su uso en el tratamiento de enfermedades por bunyavirus y togavirus |
| CA2772813A1 (en) * | 2009-09-04 | 2011-03-10 | The Chancellor, Masters And Scholars Of The University Of Oxford | Iminosugars and methods of treating filoviral diseases |
| JP5653438B2 (ja) * | 2009-09-04 | 2015-01-14 | ユナイテッド セラピューティクス コーポレイション | オルトミクソウイルス感染の治療方法 |
| CA2772807A1 (en) * | 2009-09-04 | 2011-03-10 | The Chancellor, Masters And Scholars Of The University Of Oxford | Methods of treating poxviral infections |
| CA3140959A1 (en) | 2009-11-27 | 2011-06-03 | Genzyme Corporation | An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase |
| US8961959B2 (en) | 2011-10-17 | 2015-02-24 | The Regents Of The University Of Michigan | Glucosylceramide synthase inhibitors and therapeutic methods using the same |
| WO2014017915A2 (en) | 2012-07-27 | 2014-01-30 | Universiteit Utrecht Holding B.V. | Urea and guanidinium derivatives of iminosugars |
| CN106102464A (zh) * | 2013-05-02 | 2016-11-09 | 牛津大学之校长及学者 | 采用亚氨基糖的糖脂抑制 |
| WO2016126572A2 (en) | 2015-02-02 | 2016-08-11 | The Regents Of The University Of Michigan | Glucosylceramide synthase inhibitors and therapeutic methods using the same |
| EP3318277A1 (en) | 2016-11-04 | 2018-05-09 | Institut du Cerveau et de la Moelle Epiniere-ICM | Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases |
| WO2020193746A1 (en) | 2019-03-28 | 2020-10-01 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for predicting the survival of patients suffering from melanoma |
| EP4048397B1 (en) | 2019-10-23 | 2023-08-16 | The Regents Of The University Of Michigan | Glucosylceramide synthase inhibitors for therapeutic applications |
| EP4142878B1 (en) | 2020-04-28 | 2024-06-26 | The Regents of The University of Michigan | Pyridine inhibitors of glucosylceramide synthase and therapeutic methods using the same |
| KR102826412B1 (ko) * | 2022-11-01 | 2025-06-26 | 차의과학대학교 산학협력단 | N-노닐데옥시노지리마이신을 포함하는 피부 가려움증의 억제, 경감, 개선, 또는 치료를 위한 조성물 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS61200967A (ja) * | 1985-02-28 | 1986-09-05 | バイエル・アクチエンゲゼルシヤフト | ３，４，５‐トリヒドロキシピペリジン類、その製法および用途 |
| WO1998002161A1 (en) * | 1996-07-15 | 1998-01-22 | Universiteit Van Amsterdam | Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5030638A (en) | 1990-02-26 | 1991-07-09 | G. D. Searle & Co. | Method of antiviral enhancement |
| US5798366A (en) | 1993-05-13 | 1998-08-25 | Monsanto Company | Method for treatment of CNS-involved lysosomal storage diseases |
| US5399567A (en) | 1993-05-13 | 1995-03-21 | Monsanto Company | Method of treating cholera |
| US6291657B1 (en) | 1993-05-13 | 2001-09-18 | Monsanto Company | Deoxygalactonojirimycin derivatives |
| CA2252238A1 (en) | 1997-10-31 | 1999-04-30 | Sun Microsystems, Inc. | Method and apparatus for sharing a time quantum |
-
1999
- 1999-12-02 US US09/453,754 patent/US6610703B1/en not_active Expired - Fee Related
- 1999-12-08 ES ES99967135T patent/ES2190674T3/es not_active Expired - Lifetime
- 1999-12-08 WO PCT/US1999/027918 patent/WO2000033843A1/en not_active Ceased
- 1999-12-08 AU AU23475/00A patent/AU2347500A/en not_active Abandoned
- 1999-12-08 EP EP99967135A patent/EP1137416B1/en not_active Expired - Lifetime
- 1999-12-08 PT PT99967135T patent/PT1137416E/pt unknown
- 1999-12-08 AT AT99967135T patent/ATE226821T1/de not_active IP Right Cessation
- 1999-12-08 DK DK99967135T patent/DK1137416T3/da active
- 1999-12-08 DE DE69903750T patent/DE69903750T2/de not_active Expired - Fee Related
- 1999-12-08 JP JP2000586335A patent/JP2002531505A/ja active Pending
- 1999-12-08 CA CA002352544A patent/CA2352544C/en not_active Expired - Fee Related
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS61200967A (ja) * | 1985-02-28 | 1986-09-05 | バイエル・アクチエンゲゼルシヤフト | ３，４，５‐トリヒドロキシピペリジン類、その製法および用途 |
| WO1998002161A1 (en) * | 1996-07-15 | 1998-01-22 | Universiteit Van Amsterdam | Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors |
Also Published As
| Publication number | Publication date |
|---|---|
| DE69903750D1 (de) | 2002-12-05 |
| EP1137416B1 (en) | 2002-10-30 |
| EP1137416A1 (en) | 2001-10-04 |
| PT1137416E (pt) | 2003-01-31 |
| US6610703B1 (en) | 2003-08-26 |
| DE69903750T2 (de) | 2003-06-12 |
| CA2352544C (en) | 2007-11-13 |
| ES2190674T3 (es) | 2003-08-01 |
| CA2352544A1 (en) | 2000-06-15 |
| AU2347500A (en) | 2000-06-26 |
| DK1137416T3 (da) | 2003-03-03 |
| WO2000033843A1 (en) | 2000-06-15 |
| ATE226821T1 (de) | 2002-11-15 |
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