JP2016503405A5 - - Google Patents
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- Publication number
- JP2016503405A5 JP2016503405A5 JP2015540845A JP2015540845A JP2016503405A5 JP 2016503405 A5 JP2016503405 A5 JP 2016503405A5 JP 2015540845 A JP2015540845 A JP 2015540845A JP 2015540845 A JP2015540845 A JP 2015540845A JP 2016503405 A5 JP2016503405 A5 JP 2016503405A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- aav
- disease
- dementia
- 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.)
- Abandoned
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261722434P | 2012-11-05 | 2012-11-05 | |
| US61/722,434 | 2012-11-05 | ||
| PCT/US2013/068242 WO2014071282A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016503405A JP2016503405A (ja) | 2016-02-04 |
| JP2016503405A5 true JP2016503405A5 (enExample) | 2016-12-22 |
Family
ID=49578599
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015540845A Abandoned JP2016503405A (ja) | 2012-11-05 | 2013-11-04 | タンパク質症を処置するための組成物および方法 |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20150284472A1 (enExample) |
| EP (1) | EP2914281A1 (enExample) |
| JP (1) | JP2016503405A (enExample) |
| KR (1) | KR20150079751A (enExample) |
| CN (1) | CN104902923A (enExample) |
| AR (1) | AR093355A1 (enExample) |
| AU (1) | AU2013337354A1 (enExample) |
| BR (1) | BR112015009746A2 (enExample) |
| CA (1) | CA2889990A1 (enExample) |
| CL (1) | CL2015001157A1 (enExample) |
| CR (1) | CR20150216A (enExample) |
| EA (1) | EA201590880A1 (enExample) |
| HK (1) | HK1214521A1 (enExample) |
| IL (1) | IL238416A0 (enExample) |
| MA (1) | MA38144A1 (enExample) |
| MX (1) | MX2015005722A (enExample) |
| PH (1) | PH12015500879A1 (enExample) |
| SG (1) | SG11201502989XA (enExample) |
| TN (1) | TN2015000171A1 (enExample) |
| TW (1) | TW201427695A (enExample) |
| WO (1) | WO2014071282A1 (enExample) |
| ZA (1) | ZA201502618B (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2484371B1 (en) | 2008-06-26 | 2014-12-17 | Orphazyme APS | Use of Hsp70 as a regulator of enzymatic activity |
| RU2013125923A (ru) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| US10709700B2 (en) | 2014-09-15 | 2020-07-14 | Orphazyme A/S | Arimoclomol formulation |
| WO2016179497A1 (en) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| SG10202100652VA (en) * | 2015-07-21 | 2021-03-30 | Univ Jefferson | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| BR112018014870A2 (pt) * | 2016-01-21 | 2018-12-11 | Protein Dynamic Solutions Llc | método e sistema para análise de dados espectrais |
| EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| SI3448382T1 (sl) | 2016-04-29 | 2021-03-31 | Orphazyme A/S, c/o COBIS A/S | Arimoclomol za zdravljenje motenj, povezanih z glukocerebrozidazo |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| CN109715194B (zh) * | 2016-08-03 | 2024-06-11 | 南佛罗里达大学 | 用于治疗神经障碍的颤蛋白组合物 |
| BR112020006633A2 (pt) * | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| CN112553210B (zh) * | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN111819281B (zh) * | 2017-10-23 | 2024-12-31 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
| KR20240171186A (ko) * | 2017-10-26 | 2024-12-06 | 다케다 야쿠힌 고교 가부시키가이샤 | 글루코세레브로시다제 및 이소파고민을 포함하는 제제 |
| US20210041461A1 (en) * | 2018-03-14 | 2021-02-11 | Yissum Research Developmen Company Of The Hebrew University Of Jerusalem Ltd | Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies |
| KR20220006527A (ko) * | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| US11999974B2 (en) | 2019-04-10 | 2024-06-04 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | Agents for reversing toxic proteinopathies |
| TW202214864A (zh) | 2020-08-06 | 2022-04-16 | 應用醫學研究基金會 | 用於藉基因療法治療諸如阿茲海默症之Ｔａｕ蛋白病變的病毒顆粒 |
| WO2022066911A1 (en) * | 2020-09-23 | 2022-03-31 | Seelos Therapeutics, Inc. | Compositions and methods for inhibiting alpha-synuclein aggregation |
| JP2023545462A (ja) * | 2020-10-14 | 2023-10-30 | デナリ セラピューティクス インコーポレイテッド | 前頭側頭型認知症を治療及びモニタリングするための方法 |
| WO2022106614A1 (en) | 2020-11-19 | 2022-05-27 | Orphazyme A/S | Processes for preparing arimoclomol citrate and intermediates thereof |
| CN112569354B (zh) * | 2020-12-29 | 2022-06-10 | 四川大学华西医院 | tau蛋白及其基因作为药物靶点在制备治疗糖尿病药物中的应用 |
| JP2024546887A (ja) * | 2021-12-17 | 2024-12-26 | ジェネンテック， インコーポレイテッド | オリゴヌクレオチドｇｂａアゴニスト |
| EP4448762A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotides capable of increasing glucocerebrosidase expression |
Family Cites Families (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4816567A (en) | 1983-04-08 | 1989-03-28 | Genentech, Inc. | Recombinant immunoglobin preparations |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US4676980A (en) | 1985-09-23 | 1987-06-30 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Target specific cross-linked heteroantibodies |
| US5225539A (en) | 1986-03-27 | 1993-07-06 | Medical Research Council | Recombinant altered antibodies and methods of making altered antibodies |
| US5892019A (en) | 1987-07-15 | 1999-04-06 | The United States Of America, As Represented By The Department Of Health And Human Services | Production of a single-gene-encoded immunoglobulin |
| GB8823869D0 (en) | 1988-10-12 | 1988-11-16 | Medical Res Council | Production of antibodies |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| US6172197B1 (en) | 1991-07-10 | 2001-01-09 | Medical Research Council | Methods for producing members of specific binding pairs |
| US5633425A (en) | 1990-08-29 | 1997-05-27 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
| AU8507191A (en) | 1990-08-29 | 1992-03-30 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
| US5625126A (en) | 1990-08-29 | 1997-04-29 | Genpharm International, Inc. | Transgenic non-human animals for producing heterologous antibodies |
| US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
| US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
| ES2113940T3 (es) | 1990-12-03 | 1998-05-16 | Genentech Inc | Metodo de enriquecimiento para variantes de proteinas con propiedades de union alteradas. |
| PT1696031E (pt) | 1991-12-02 | 2010-06-25 | Medical Res Council | Produção de auto-anticorpos a partir de reportórios de segmentos de anticorpo e exibidos em fagos |
| ATE304604T1 (de) | 1993-06-24 | 2005-09-15 | Frank L Graham | Adenovirus vektoren für gentherapie |
| DE69434594T2 (de) | 1993-10-25 | 2006-09-21 | Canji, Inc., San Diego | Rekombinante adenoviren-vektor und verfahren zur verwendung |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| ATE219517T1 (de) | 1995-08-18 | 2002-07-15 | Morphosys Ag | Protein-/(poly)peptidbibliotheken |
| US6696248B1 (en) | 1995-08-18 | 2004-02-24 | Morphosys Ag | Protein/(poly)peptide libraries |
| US6924123B2 (en) | 1996-10-29 | 2005-08-02 | Oxford Biomedica (Uk) Limited | Lentiviral LTR-deleted vector |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ATE482275T1 (de) | 1999-07-02 | 2010-10-15 | Morphosys Ag | Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| AU2001269923A1 (en) | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US7446098B2 (en) * | 2003-02-18 | 2008-11-04 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiencies |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| ATE555788T1 (de) * | 2006-06-23 | 2012-05-15 | Amicus Therapeutics Inc | Verfahren zur behandlung neurologischer erkrankungen mittels verstärkung der beta - glucocerebrosidaseaktivität |
| US8454954B2 (en) * | 2007-05-16 | 2013-06-04 | The Brigham And Women's Hospital, Inc. | Treatment of synucleinopathies |
| US9845327B2 (en) | 2011-06-22 | 2017-12-19 | The General Hospital Corporation | Treatment of proteinopathies |
| AU2013240187A1 (en) | 2012-03-28 | 2014-10-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Salicylic acid derivatives useful as glucocerebrosidase activators |
-
2013
- 2013-11-04 BR BR112015009746A patent/BR112015009746A2/pt not_active IP Right Cessation
- 2013-11-04 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Withdrawn
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en not_active Ceased
- 2013-11-04 HK HK16102536.9A patent/HK1214521A1/zh unknown
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-05 AR ARP130104033A patent/AR093355A1/es unknown
-
2015
- 2015-04-17 ZA ZA2015/02618A patent/ZA201502618B/en unknown
- 2015-04-20 PH PH12015500879A patent/PH12015500879A1/en unknown
- 2015-04-21 IL IL238416A patent/IL238416A0/en unknown
- 2015-04-28 CR CR20150216A patent/CR20150216A/es unknown
- 2015-04-30 CL CL2015001157A patent/CL2015001157A1/es unknown
- 2015-05-04 TN TNP2015000171A patent/TN2015000171A1/fr unknown
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