JP2009541489A5 - - Google Patents
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- Publication number
- JP2009541489A5 JP2009541489A5 JP2009518489A JP2009518489A JP2009541489A5 JP 2009541489 A5 JP2009541489 A5 JP 2009541489A5 JP 2009518489 A JP2009518489 A JP 2009518489A JP 2009518489 A JP2009518489 A JP 2009518489A JP 2009541489 A5 JP2009541489 A5 JP 2009541489A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmacological chaperone
- glucocerebrosidase
- isophagomine
- disease
- individual
- 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.)
- Granted
Links
- 108010006519 Molecular Chaperones Proteins 0.000 claims 15
- 230000000144 pharmacologic effect Effects 0.000 claims 15
- 102000004547 Glucosylceramidase Human genes 0.000 claims 11
- 108010017544 Glucosylceramidase Proteins 0.000 claims 11
- 125000000217 alkyl group Chemical group 0.000 claims 6
- -1 isofagomine compound Chemical class 0.000 claims 6
- 230000004770 neurodegeneration Effects 0.000 claims 6
- 208000015122 neurodegenerative disease Diseases 0.000 claims 6
- 229910052760 oxygen Inorganic materials 0.000 claims 6
- 229910052717 sulfur Inorganic materials 0.000 claims 6
- 230000000694 effects Effects 0.000 claims 5
- 208000032859 Synucleinopathies Diseases 0.000 claims 4
- QPYJXFZUIJOGNX-HSUXUTPPSA-N afegostat Chemical class OC[C@H]1CNC[C@@H](O)[C@@H]1O QPYJXFZUIJOGNX-HSUXUTPPSA-N 0.000 claims 4
- 229910052799 carbon Inorganic materials 0.000 claims 4
- 229910052739 hydrogen Inorganic materials 0.000 claims 4
- 239000001257 hydrogen Substances 0.000 claims 4
- 229910052757 nitrogen Inorganic materials 0.000 claims 4
- 208000009829 Lewy Body Disease Diseases 0.000 claims 3
- 201000002832 Lewy body dementia Diseases 0.000 claims 3
- 208000018737 Parkinson disease Diseases 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000003112 inhibitor Substances 0.000 claims 3
- ULBPPCHRAVUQMC-MUMXBIPUSA-N (2r,3r)-2,3-dihydroxybutanedioic acid;(3r,4r,5r)-5-(hydroxymethyl)piperidine-3,4-diol Chemical group OC[C@H]1CNC[C@@H](O)[C@@H]1O.OC(=O)[C@H](O)[C@@H](O)C(O)=O ULBPPCHRAVUQMC-MUMXBIPUSA-N 0.000 claims 2
- 125000006201 3-phenylpropyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])([H])C([H])([H])* 0.000 claims 2
- 102000006378 Catechol O-methyltransferase Human genes 0.000 claims 2
- 108020002739 Catechol O-methyltransferase Proteins 0.000 claims 2
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 2
- 208000001089 Multiple system atrophy Diseases 0.000 claims 2
- 125000004946 alkenylalkyl group Chemical group 0.000 claims 2
- 125000004183 alkoxy alkyl group Chemical group 0.000 claims 2
- 125000003545 alkoxy group Chemical group 0.000 claims 2
- 125000003118 aryl group Chemical group 0.000 claims 2
- 125000005242 carbamoyl alkyl group Chemical group 0.000 claims 2
- 230000002860 competitive effect Effects 0.000 claims 2
- 150000001875 compounds Chemical class 0.000 claims 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 125000001188 haloalkyl group Chemical group 0.000 claims 2
- 125000001475 halogen functional group Chemical group 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 150000002431 hydrogen Chemical class 0.000 claims 2
- 229910052738 indium Inorganic materials 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 229940098778 Dopamine receptor agonist Drugs 0.000 claims 1
- WTDRDQBEARUVNC-LURJTMIESA-N L-DOPA Chemical group OC(=O)[C@@H](N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-LURJTMIESA-N 0.000 claims 1
- WTDRDQBEARUVNC-UHFFFAOYSA-N L-Dopa Natural products OC(=O)C(N)CC1=CC=C(O)C(O)=C1 WTDRDQBEARUVNC-UHFFFAOYSA-N 0.000 claims 1
- 229940123685 Monoamine oxidase inhibitor Drugs 0.000 claims 1
- 229940121948 Muscarinic receptor antagonist Drugs 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 102000003802 alpha-Synuclein Human genes 0.000 claims 1
- 108090000185 alpha-Synuclein Proteins 0.000 claims 1
- 239000002260 anti-inflammatory agent Substances 0.000 claims 1
- 229940121363 anti-inflammatory agent Drugs 0.000 claims 1
- 239000000812 cholinergic antagonist Substances 0.000 claims 1
- 239000003954 decarboxylase inhibitor Substances 0.000 claims 1
- 239000003136 dopamine receptor stimulating agent Substances 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 229960004502 levodopa Drugs 0.000 claims 1
- 239000002899 monoamine oxidase inhibitor Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81595206P | 2006-06-23 | 2006-06-23 | |
| US60/815,952 | 2006-06-23 | ||
| PCT/US2007/072016 WO2007150064A2 (en) | 2006-06-23 | 2007-06-25 | METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2009541489A JP2009541489A (ja) | 2009-11-26 |
| JP2009541489A5 true JP2009541489A5 (enExample) | 2010-08-12 |
| JP5303458B2 JP5303458B2 (ja) | 2013-10-02 |
Family
ID=38834440
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2009518489A Active JP5303458B2 (ja) | 2006-06-23 | 2007-06-25 | 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 |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US7829579B2 (enExample) |
| EP (1) | EP2040548B1 (enExample) |
| JP (1) | JP5303458B2 (enExample) |
| KR (1) | KR101402554B1 (enExample) |
| AT (1) | ATE555788T1 (enExample) |
| AU (1) | AU2007260812B2 (enExample) |
| BR (1) | BRPI0713442A2 (enExample) |
| CA (1) | CA2656643C (enExample) |
| DK (1) | DK2040548T3 (enExample) |
| ES (1) | ES2387845T3 (enExample) |
| IL (1) | IL196151A (enExample) |
| MX (1) | MX2009000032A (enExample) |
| PT (1) | PT2040548E (enExample) |
| WO (1) | WO2007150064A2 (enExample) |
| ZA (1) | ZA200900270B (enExample) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| 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 |
| GB0622702D0 (en) * | 2006-11-15 | 2006-12-27 | Univ Dundee | Selective glycosidase inhibitors |
| HUE027278T2 (en) | 2007-05-16 | 2016-11-28 | Brigham & Womens Hospital Inc | Treatment of synucleinopathies |
| ES2963910T3 (es) | 2008-06-26 | 2024-04-03 | Zevra Denmark As | Uso de Hsp70 como regulador de la actividad enzimática |
| BRPI0920498A2 (pt) * | 2008-11-14 | 2019-09-24 | Neuraltus Pharmaceuticals Inc | método de tratamento de uma condição em um sujeito |
| GB0906159D0 (en) * | 2009-04-09 | 2009-05-20 | Summit Corp Plc | Drug combination for the treatment of proteostatic diseases |
| JP5634498B2 (ja) * | 2009-04-09 | 2014-12-03 | 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の予防および／または治療方法 |
| CA2758271C (en) * | 2009-04-09 | 2018-06-05 | Robert Boyd | Methods for preventing and/or treating lysosomal storage disorders |
| US9206457B2 (en) * | 2009-05-26 | 2015-12-08 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| US20110070643A1 (en) * | 2009-05-26 | 2011-03-24 | Do Hung V | Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics |
| NZ598156A (en) * | 2009-07-28 | 2014-06-27 | Shire Human Genetic Therapies | Compositions and methods for treating gaucher disease |
| KR101880411B1 (ko) * | 2009-10-19 | 2018-07-19 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 중추신경계의 퇴행성 질환을 예방 및/또는 치료하는 신규 조성물 |
| PL2490712T3 (pl) | 2009-10-19 | 2015-12-31 | Amicus Therapeutics Inc | Sposób leczenia choroby Alzheimera przy zastosowaniu chaperonów farmakologicznych dla zwiększenia aktywności gangliozydaz |
| CN102647905B (zh) * | 2009-10-19 | 2015-09-09 | 阿米库斯治疗学公司 | 用于预防和/或治疗溶酶体贮积失调的新颖组合物 |
| PT2646044T (pt) | 2010-11-30 | 2019-11-12 | Orphazyme As | Métodos para aumentar a atividade intracelular de hsp70 |
| PT2707101T (pt) | 2011-05-12 | 2019-05-30 | Proteostasis Therapeutics Inc | Reguladores da proteostase |
| EP2533051A1 (en) * | 2011-06-06 | 2012-12-12 | Centogene GmbH | Method for the diagnosis of Gaucher's disease |
| WO2012177997A1 (en) | 2011-06-22 | 2012-12-27 | The General Hospital Corporation | Treatment of proteinopathies |
| CA2861464C (en) | 2012-01-25 | 2018-03-06 | Proteostasis Therapeutics, Inc. | Proteasome activity modulating tricyclic compounds |
| BR112015009746A2 (pt) * | 2012-11-05 | 2017-08-15 | Genzyme Corp | Agente que aumenta atividade de glicocerebrosidase em mamíferos |
| WO2014089449A1 (en) | 2012-12-07 | 2014-06-12 | Rush University Nedical Center | Composition and method for treating neuronal ceroid lipofuscinosis |
| EP3087056A4 (en) | 2013-12-23 | 2017-07-19 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
| US9675627B2 (en) | 2014-04-14 | 2017-06-13 | Amicus Therapeutics, Inc. | Dosing regimens for treating and/or preventing cerebral amyloidoses |
| HUE054957T2 (hu) | 2014-09-15 | 2021-10-28 | Orphazyme As | Arimoklomol készítése |
| SMT202400187T1 (it) | 2014-11-06 | 2024-07-09 | Bial R&D Investments S A | Pirazolo(1,5-a)pirimidine sostituite e loro utilizzo nel trattamento di disturbi medici |
| EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| AU2017246455B2 (en) | 2016-04-06 | 2021-09-30 | Bial - R&D Investments, S.A. | Pyrazolo[1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders |
| US10787454B2 (en) | 2016-04-06 | 2020-09-29 | BIAL—BioTech Investments, Inc. | Imidazo[1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders |
| WO2017176962A1 (en) * | 2016-04-06 | 2017-10-12 | Lysosomal Therapeutics, Inc. | Pyrrolo[1,2-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| RU2021117465A (ru) | 2016-04-29 | 2021-07-22 | Орфазим А/С | Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений |
| BR112018072552A8 (pt) | 2016-05-05 | 2023-03-14 | Lysosomal Therapeutics Inc | Compostos de imidazo [1,2-b]piridazinas e imidazo [1,5-b] piridazinas substituídas, composição farmacêutica que os compreende e seu uso no tratamento de distúrbios médicos |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| KR20240171186A (ko) * | 2017-10-26 | 2024-12-06 | 다케다 야쿠힌 고교 가부시키가이샤 | 글루코세레브로시다제 및 이소파고민을 포함하는 제제 |
| CN108428452B (zh) | 2018-03-14 | 2019-12-13 | 百度在线网络技术（北京）有限公司 | 终端支架和远场语音交互系统 |
| WO2020006269A1 (en) | 2018-06-27 | 2020-01-02 | Proteostasis Therapeutics, Inc. | Proteasome activity enhancing compounds |
| BR112020026580A2 (pt) | 2018-06-27 | 2021-03-23 | Proteostasis Therapeutics, Inc. (Fem) | Compostos, composição farmacêutica e métodos para tratar um paciente |
| 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 |
| WO2020118056A1 (en) * | 2018-12-05 | 2020-06-11 | Washington University | Methods of detecting, preventing, reversing, and treating neurological diseases |
| US12286400B2 (en) | 2019-04-25 | 2025-04-29 | Takeda Pharmaceutical Company Limited | Isofagomine salts, methods of use and formulations |
| CN111450306B (zh) * | 2020-03-06 | 2021-11-19 | 大连理工大学 | 外用纳米羟基磷灰石/聚多巴胺湿黏附型止血粉及其制备方法 |
| IL303026A (en) | 2020-11-19 | 2023-07-01 | Zevra Denmark As | Processes for preparing arimoclomol citrate and intermediates thereof |
| WO2022232383A1 (en) * | 2021-04-30 | 2022-11-03 | Vanqua Bio, Inc. | Small molecule modulators of glucocerebrosidase activity and uses thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0934531B1 (en) * | 1996-10-15 | 2004-08-04 | D-Gen Limited | Diagnosis of spongiform encephalopathy |
| GB2355074A (en) | 1996-10-15 | 2001-04-11 | Imperial College | Method for predicting susceptibility to bovine spongiform encephalopathy |
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| WO2004069190A2 (en) * | 2003-01-31 | 2004-08-19 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| BRPI0416216A (pt) | 2003-11-12 | 2006-12-26 | Amicus Therapeutics Inc | composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina |
| MX2007015602A (es) | 2005-06-08 | 2008-02-21 | Amicus Therapeutics Inc | Tratamiento de trastornos del sistema nervioso central asociados con mutaciones en genes que codifican las enzimas lisosomicas. |
| MX2007006175A (es) * | 2006-05-24 | 2009-02-16 | Amicus Therapeutics Inc | Sal de tartrato de isofagomina y metodos de uso. |
| 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 |
| KR101650726B1 (ko) | 2007-05-22 | 2016-08-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 이소파고민과 이의 유도체를 제조하는 새로운 방법 |
-
2007
- 2007-06-25 JP JP2009518489A patent/JP5303458B2/ja active Active
- 2007-06-25 AT AT07812295T patent/ATE555788T1/de active
- 2007-06-25 PT PT07812295T patent/PT2040548E/pt unknown
- 2007-06-25 CA CA2656643A patent/CA2656643C/en not_active Expired - Fee Related
- 2007-06-25 WO PCT/US2007/072016 patent/WO2007150064A2/en not_active Ceased
- 2007-06-25 BR BRPI0713442-8A patent/BRPI0713442A2/pt not_active Application Discontinuation
- 2007-06-25 MX MX2009000032A patent/MX2009000032A/es active IP Right Grant
- 2007-06-25 DK DK07812295T patent/DK2040548T3/da active
- 2007-06-25 US US11/768,043 patent/US7829579B2/en active Active
- 2007-06-25 ES ES07812295T patent/ES2387845T3/es active Active
- 2007-06-25 KR KR1020097001465A patent/KR101402554B1/ko not_active Expired - Fee Related
- 2007-06-25 EP EP20070812295 patent/EP2040548B1/en active Active
- 2007-06-25 AU AU2007260812A patent/AU2007260812B2/en active Active
-
2008
- 2008-12-23 IL IL196151A patent/IL196151A/en active IP Right Grant
-
2009
- 2009-01-13 ZA ZA2009/00270A patent/ZA200900270B/en unknown
-
2010
- 2010-11-08 US US12/941,468 patent/US9119845B2/en active Active
-
2014
- 2014-08-26 US US14/469,008 patent/US9402837B2/en active Active
-
2016
- 2016-08-01 US US15/225,029 patent/US10064851B2/en active Active
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