JP2014526904A5 - - Google Patents
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- JP2014526904A5 JP2014526904A5 JP2014528515A JP2014528515A JP2014526904A5 JP 2014526904 A5 JP2014526904 A5 JP 2014526904A5 JP 2014528515 A JP2014528515 A JP 2014528515A JP 2014528515 A JP2014528515 A JP 2014528515A JP 2014526904 A5 JP2014526904 A5 JP 2014526904A5
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- JP
- Japan
- Prior art keywords
- compound
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- disease
- subject
- ppca
- 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
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- 102100022166 E3 ubiquitin-protein ligase NEURL1 Human genes 0.000 claims description 7
- 102100028524 Lysosomal protective protein Human genes 0.000 claims description 6
- 230000000694 effects Effects 0.000 claims description 4
- 101001123859 Homo sapiens Sialidase-1 Proteins 0.000 claims description 2
- 150000001875 compounds Chemical class 0.000 claims 17
- 208000024827 Alzheimer disease Diseases 0.000 claims 5
- 101000973232 Homo sapiens E3 ubiquitin-protein ligase NEURL1 Proteins 0.000 claims 4
- 239000012634 fragment Substances 0.000 claims 4
- 230000004770 neurodegeneration Effects 0.000 claims 3
- 208000015122 neurodegenerative disease Diseases 0.000 claims 3
- 102100028760 Sialidase-1 Human genes 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 230000028023 exocytosis Effects 0.000 claims 2
- ASUTZQLVASHGKV-JDFRZJQESA-N galanthamine Chemical compound O1C(=C23)C(OC)=CC=C2CN(C)CC[C@]23[C@@H]1C[C@@H](O)C=C2 ASUTZQLVASHGKV-JDFRZJQESA-N 0.000 claims 2
- 230000002132 lysosomal effect Effects 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 102000005572 Cathepsin A Human genes 0.000 claims 1
- 108010059081 Cathepsin A Proteins 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- XSVMFMHYUFZWBK-NSHDSACASA-N Rivastigmine Chemical compound CCN(C)C(=O)OC1=CC=CC([C@H](C)N(C)C)=C1 XSVMFMHYUFZWBK-NSHDSACASA-N 0.000 claims 1
- 101710128612 Sialidase-1 Proteins 0.000 claims 1
- 230000007423 decrease Effects 0.000 claims 1
- ADEBPBSSDYVVLD-UHFFFAOYSA-N donepezil Chemical group O=C1C=2C=C(OC)C(OC)=CC=2CC1CC(CC1)CCN1CC1=CC=CC=C1 ADEBPBSSDYVVLD-UHFFFAOYSA-N 0.000 claims 1
- 230000006870 function Effects 0.000 claims 1
- 229960003980 galantamine Drugs 0.000 claims 1
- ASUTZQLVASHGKV-UHFFFAOYSA-N galanthamine hydrochloride Natural products O1C(=C23)C(OC)=CC=C2CN(C)CCC23C1CC(O)C=C2 ASUTZQLVASHGKV-UHFFFAOYSA-N 0.000 claims 1
- BUGYDGFZZOZRHP-UHFFFAOYSA-N memantine Chemical compound C1C(C2)CC3(C)CC1(C)CC2(N)C3 BUGYDGFZZOZRHP-UHFFFAOYSA-N 0.000 claims 1
- 229960004640 memantine Drugs 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000001681 protective effect Effects 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 229960004136 rivastigmine Drugs 0.000 claims 1
- 229960001685 tacrine Drugs 0.000 claims 1
- YLJREFDVOIBQDA-UHFFFAOYSA-N tacrine Chemical compound C1=CC=C2C(N)=C(CCCC3)C3=NC2=C1 YLJREFDVOIBQDA-UHFFFAOYSA-N 0.000 claims 1
- 101710162021 Lysosomal protective protein Proteins 0.000 description 3
- 101150024252 Neu1 gene Proteins 0.000 description 3
- 238000011818 5xFAD mouse Methods 0.000 description 2
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 108010006519 Molecular Chaperones Proteins 0.000 description 1
- 108700019146 Transgenes Proteins 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 230000000971 hippocampal effect Effects 0.000 description 1
- 102000046216 human NEU1 Human genes 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 230000003834 intracellular effect Effects 0.000 description 1
- 230000002459 sustained effect Effects 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161529675P | 2011-08-31 | 2011-08-31 | |
| US61/529,675 | 2011-08-31 | ||
| US201161544855P | 2011-10-07 | 2011-10-07 | |
| US61/544,855 | 2011-10-07 | ||
| PCT/US2012/052629 WO2013033074A2 (en) | 2011-08-31 | 2012-08-28 | Methods and compositions to detect the level of lysosomal exocytosis activity and methods of use |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014526904A JP2014526904A (ja) | 2014-10-09 |
| JP2014526904A5 true JP2014526904A5 (cg-RX-API-DMAC10.html) | 2017-06-29 |
| JP6228121B2 JP6228121B2 (ja) | 2017-11-08 |
Family
ID=46881159
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014528515A Active JP6228121B2 (ja) | 2011-08-31 | 2012-08-28 | リソソームエキソサイトーシス活性のレベルを検出する方法及び組成物並びに使用方法 |
Country Status (15)
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2011001341A (es) | 2008-08-19 | 2011-03-29 | Xenoport Inc | Prodrogas de metil hidrogeno fumarato, sus composiciones farmaceuticas, y metodos de uso. |
| DK2751279T3 (da) * | 2011-08-31 | 2017-11-20 | St Jude Children's Res Hospital | Fremgangsmåder og sammensætninger til påvisning af niveauet af lysosomal exocytose-aktivitet og fremgangsmåder til anvendelse |
| EP2887934A1 (en) | 2012-08-22 | 2015-07-01 | XenoPort, Inc. | Methods of administering monomethyl fumarate and prodrugs thereof having reduced side effects |
| US9597292B2 (en) | 2012-08-22 | 2017-03-21 | Xenoport, Inc. | Oral dosage forms of methyl hydrogen fumarate and prodrugs thereof |
| US10179118B2 (en) | 2013-03-24 | 2019-01-15 | Arbor Pharmaceuticals, Llc | Pharmaceutical compositions of dimethyl fumarate |
| WO2014197860A1 (en) | 2013-06-07 | 2014-12-11 | Xenoport, Inc. | Method of making monomethyl fumarate |
| US9421182B2 (en) | 2013-06-21 | 2016-08-23 | Xenoport, Inc. | Cocrystals of dimethyl fumarate |
| JP2016534133A (ja) | 2013-09-06 | 2016-11-04 | ゼノポート，インコーポレイティド | （ｎ，ｎ−ジエチルカルバモイル）メチル メチル（２ｅ）ブト−２−エン−１，４−ジオエートの結晶形態、その合成方法及び使用 |
| US9999672B2 (en) | 2014-03-24 | 2018-06-19 | Xenoport, Inc. | Pharmaceutical compositions of fumaric acid esters |
| WO2016179481A1 (en) * | 2015-05-07 | 2016-11-10 | Cardelli James Allen | Cancer treatment via repositioned tricyclic anti-depressant-like drugs as anti-cancer agents and new combinations of such drugs |
| AU2016343812A1 (en) * | 2015-10-30 | 2018-05-10 | Ultragenyx Pharmaceutical Inc. | Methods and compositions for the treatment of amyloidosis |
| AU2017233072B2 (en) * | 2016-03-18 | 2020-12-24 | Nantcell, Inc. | Multimodal vector for dendritic cell infection |
| DK3366063T3 (da) * | 2017-01-04 | 2020-04-27 | Ericsson Telefon Ab L M | Fremgangsmåde, basisstation og brugerindretning til at sende/modtage systeminformation |
| CN114555800A (zh) | 2019-07-05 | 2022-05-27 | 国立大学法人德岛大学 | 经修饰的神经氨酸酶 |
| JP2025504704A (ja) * | 2022-01-31 | 2025-02-14 | センス リサーチ ファウンデーション | 老化細胞表面マーカー |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4769331A (en) | 1981-09-16 | 1988-09-06 | University Patents, Inc. | Recombinant methods and materials |
| NZ201918A (en) | 1981-09-18 | 1987-04-30 | Genentech Inc | N-terminal methionyl analogues of bovine growth hormone |
| US5288641A (en) | 1984-06-04 | 1994-02-22 | Arch Development Corporation | Herpes Simplex virus as a vector |
| US4859587A (en) | 1984-06-04 | 1989-08-22 | Institut Merieux | Recombinant herpes simplex viruses, vaccines and methods |
| US4925673A (en) | 1986-08-18 | 1990-05-15 | Clinical Technologies Associates, Inc. | Delivery systems for pharmacological agents encapsulated with proteinoids |
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US5013556A (en) | 1989-10-20 | 1991-05-07 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5328688A (en) | 1990-09-10 | 1994-07-12 | Arch Development Corporation | Recombinant herpes simplex viruses vaccines and methods |
| US5605793A (en) | 1994-02-17 | 1997-02-25 | Affymax Technologies N.V. | Methods for in vitro recombination |
| US5837458A (en) | 1994-02-17 | 1998-11-17 | Maxygen, Inc. | Methods and compositions for cellular and metabolic engineering |
| WO1997015588A1 (en) | 1995-10-26 | 1997-05-01 | St. Jude Children's Research Hospital | Protective protein/cathepsin a and precursor: crystallization, x-ray diffraction, three-dimensional structure determination and rational drug design |
| WO1998031817A2 (en) * | 1997-01-14 | 1998-07-23 | Hopital Sainte-Justine | Human lysosomal sialidase and therapeutic uses thereof |
| ES2752800T3 (es) * | 2000-05-01 | 2020-04-06 | Cerecin Inc | Uso de triglicéridos de cadena media para el tratamiento y la prevención de la enfermedad del Parkinson resultante de un metabolismo neuronal reducido |
| WO2001089583A2 (en) * | 2000-05-23 | 2001-11-29 | Neurologix, Inc. | Glutamic acid decarboxylase (gad)_delivery system for treating neurodegenerative diseases |
| US7232670B2 (en) | 2001-09-28 | 2007-06-19 | St. Jude Children's Research Hospital | Targeting proteins to cells expressing mannose receptors via expression in insect cells |
| WO2005042773A1 (en) | 2003-10-24 | 2005-05-12 | Gilead Sciences, Inc. | Methods and compositions for identifying therapeutic compounds |
| WO2006001877A2 (en) | 2004-04-13 | 2006-01-05 | Myriad Genetics, Inc. | Combination treatment for neurodegenerative disorders comprising r-flurbiprofen |
| WO2006013013A2 (en) * | 2004-08-04 | 2006-02-09 | Bayer Healthcare Ag | Diagnostics and therapeutics for diseases associated with protective protein for beta-galactosidase (ppgb) |
| WO2008049058A2 (en) | 2006-10-18 | 2008-04-24 | Cornell Research Foundation, Inc. | Cln2 treatment of alzheimer's disease |
| US7951367B2 (en) | 2006-11-15 | 2011-05-31 | The J. David Gladstone Institutes | Methods and compositions for reducing amyloid beta levels |
| US8420613B2 (en) | 2006-11-15 | 2013-04-16 | The J. David Gladstone Institutes | Methods and compositions for reducing amyloid beta levels |
| PL2158322T3 (pl) | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| DK2490712T3 (en) * | 2009-10-19 | 2015-10-05 | Amicus Therapeutics Inc | PROCESS FOR THE TREATMENT OF ALZHEIMER'S DISEASE BY USING Pharmacological chaperones TO INCREASE ACTIVITY OF GANGLIOSIDASER |
| US20140161896A1 (en) | 2011-08-04 | 2014-06-12 | Ramot At Tel-Aviv University Ltd. | Particles for the treatment of neurodegenerative diseases |
| DK2751279T3 (da) * | 2011-08-31 | 2017-11-20 | St Jude Children's Res Hospital | Fremgangsmåder og sammensætninger til påvisning af niveauet af lysosomal exocytose-aktivitet og fremgangsmåder til anvendelse |
-
2012
- 2012-08-28 DK DK12761845.2T patent/DK2751279T3/da active
- 2012-08-28 HR HRP20171909TT patent/HRP20171909T1/hr unknown
- 2012-08-28 ES ES12761845.2T patent/ES2653918T3/es active Active
- 2012-08-28 HU HUE12761845A patent/HUE037829T2/hu unknown
- 2012-08-28 JP JP2014528515A patent/JP6228121B2/ja active Active
- 2012-08-28 CA CA2847057A patent/CA2847057C/en active Active
- 2012-08-28 PT PT127618452T patent/PT2751279T/pt unknown
- 2012-08-28 PL PL12761845T patent/PL2751279T3/pl unknown
- 2012-08-28 EP EP12761845.2A patent/EP2751279B1/en active Active
- 2012-08-28 US US14/239,728 patent/US9399791B2/en active Active
- 2012-08-28 WO PCT/US2012/052629 patent/WO2013033074A2/en not_active Ceased
- 2012-08-28 LT LTEP12761845.2T patent/LT2751279T/lt unknown
- 2012-08-28 SI SI201231157T patent/SI2751279T1/en unknown
- 2012-08-28 AU AU2012300252A patent/AU2012300252B2/en active Active
-
2016
- 2016-06-20 US US15/187,349 patent/US9840727B2/en active Active
-
2017
- 2017-11-09 US US15/808,075 patent/US10533208B2/en active Active
- 2017-12-18 CY CY20171101321T patent/CY1119689T1/el unknown
-
2019
- 2019-11-26 US US16/696,210 patent/US20200087704A1/en not_active Abandoned
-
2022
- 2022-08-18 US US17/820,755 patent/US12640251B2/en active Active
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