AU2013337354A1 - Compositions and methods for treating proteinopathies - Google Patents
Compositions and methods for treating proteinopathies Download PDFInfo
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- AU2013337354A1 AU2013337354A1 AU2013337354A AU2013337354A AU2013337354A1 AU 2013337354 A1 AU2013337354 A1 AU 2013337354A1 AU 2013337354 A AU2013337354 A AU 2013337354A AU 2013337354 A AU2013337354 A AU 2013337354A AU 2013337354 A1 AU2013337354 A1 AU 2013337354A1
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Classifications
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- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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- C12N2750/00011—Details
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- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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Landscapes
- Health & Medical Sciences (AREA)
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- Engineering & Computer Science (AREA)
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- Molecular Biology (AREA)
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- Hospice & Palliative Care (AREA)
- Psychology (AREA)
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| US61/722,434 | 2012-11-05 | ||
| PCT/US2013/068242 WO2014071282A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
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| AU2013337354A1 true AU2013337354A1 (en) | 2015-05-21 |
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| ES2963910T3 (es) | 2008-06-26 | 2024-04-03 | Zevra Denmark As | Uso de Hsp70 como regulador de la actividad enzimática |
| PT2646044T (pt) | 2010-11-30 | 2019-11-12 | Orphazyme As | Métodos para aumentar a atividade intracelular de hsp70 |
| HUE054957T2 (hu) | 2014-09-15 | 2021-10-28 | Orphazyme As | Arimoklomol készítése |
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| SG11201901265SA (en) * | 2015-07-21 | 2019-03-28 | Univ Jefferson | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| MX2018008743A (es) * | 2016-01-21 | 2019-01-30 | Protein Dynamic Solutions Llc | Método y sistema para el análisis de datos espectrales. |
| 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 |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| AU2017306558B2 (en) * | 2016-08-03 | 2024-09-19 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| CN111465691A (zh) | 2017-10-03 | 2020-07-28 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| JP7254815B2 (ja) * | 2017-10-03 | 2023-04-10 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| 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 |
| WO2019175883A1 (en) * | 2018-03-14 | 2019-09-19 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd | Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies |
| AU2020273182B2 (en) | 2019-04-10 | 2025-11-20 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN114026115A (zh) * | 2019-04-10 | 2022-02-08 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | AGENTS TO REVERSE TOXIC PROTEINOPATHIES |
| EP4192961A2 (en) | 2020-08-06 | 2023-06-14 | Fundacion para la Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| EP4229419A2 (en) * | 2020-10-14 | 2023-08-23 | Denali Therapeutics Inc. | Methods for treating and monitoring frontotemporal dementia |
| IL303026A (en) | 2020-11-19 | 2023-07-01 | Zevra Denmark As | 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 |
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| US5225539A (en) | 1986-03-27 | 1993-07-06 | Medical Research Council | Recombinant altered antibodies and methods of making altered antibodies |
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| JP2938569B2 (ja) | 1990-08-29 | 1999-08-23 | ジェンファーム インターナショナル，インコーポレイティド | 異種免疫グロブリンを作る方法及びトランスジェニックマウス |
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| US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
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| WO1992009690A2 (en) | 1990-12-03 | 1992-06-11 | Genentech, Inc. | Enrichment method for variant proteins with altered binding properties |
| WO1993011236A1 (en) | 1991-12-02 | 1993-06-10 | Medical Research Council | Production of anti-self antibodies from antibody segment repertoires and displayed on phage |
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| ES2176484T3 (es) | 1995-08-18 | 2002-12-01 | Morphosys Ag | Bancos de proteinas/(poli)peptidos. |
| US6924123B2 (en) | 1996-10-29 | 2005-08-02 | Oxford Biomedica (Uk) Limited | Lentiviral LTR-deleted vector |
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| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | 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 |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
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| WO2001097829A2 (en) | 2000-06-19 | 2001-12-27 | 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 |
| 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 |
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- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Withdrawn
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en not_active Ceased
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 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
Also Published As
| Publication number | Publication date |
|---|---|
| BR112015009746A2 (pt) | 2017-08-15 |
| TN2015000171A1 (fr) | 2016-10-03 |
| AR093355A1 (es) | 2015-06-03 |
| MA38144A1 (fr) | 2018-08-31 |
| TW201427695A (zh) | 2014-07-16 |
| US20150284472A1 (en) | 2015-10-08 |
| CN104902923A (zh) | 2015-09-09 |
| JP2016503405A (ja) | 2016-02-04 |
| HK1214521A1 (zh) | 2016-07-29 |
| EA201590880A1 (ru) | 2015-09-30 |
| WO2014071282A1 (en) | 2014-05-08 |
| ZA201502618B (en) | 2016-01-27 |
| PH12015500879A1 (en) | 2015-06-29 |
| EP2914281A1 (en) | 2015-09-09 |
| MX2015005722A (es) | 2016-01-12 |
| CL2015001157A1 (es) | 2015-10-16 |
| CR20150216A (es) | 2015-05-29 |
| CA2889990A1 (en) | 2014-05-08 |
| SG11201502989XA (en) | 2015-05-28 |
| KR20150079751A (ko) | 2015-07-08 |
| IL238416A0 (en) | 2015-06-30 |
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