CA2889990A1 - Compositions and methods for treating proteinopathies - Google Patents
Compositions and methods for treating proteinopathies Download PDFInfo
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- CA2889990A1 CA2889990A1 CA2889990A CA2889990A CA2889990A1 CA 2889990 A1 CA2889990 A1 CA 2889990A1 CA 2889990 A CA2889990 A CA 2889990A CA 2889990 A CA2889990 A CA 2889990A CA 2889990 A1 CA2889990 A1 CA 2889990A1
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Classifications
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P25/00—Drugs for disorders of the nervous system
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
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- Engineering & Computer Science (AREA)
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- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
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- Veterinary Medicine (AREA)
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- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Neurosurgery (AREA)
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- Immunology (AREA)
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- Molecular Biology (AREA)
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- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Psychiatry (AREA)
- Plant Pathology (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| 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 (1)
| Publication Number | Publication Date |
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| CA2889990A1 true CA2889990A1 (en) | 2014-05-08 |
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Family Applications (1)
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| CA2889990A Abandoned CA2889990A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
Country Status (22)
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
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| ES2385735T3 (es) | 2008-06-26 | 2012-07-31 | Orphazyme Aps | Uso de Hsp70 como regulador de la actividad enzimática |
| HUE046235T2 (hu) | 2010-11-30 | 2020-02-28 | Orphazyme As | A HSP70 intercelluláris aktivitásának növelésére alkalmazott módszerek |
| KR20250069686A (ko) | 2014-09-15 | 2025-05-19 | 제브라 덴마크 에이/에스 | 아리모클로몰 제제 |
| US10967073B2 (en) * | 2015-05-07 | 2021-04-06 | The Mclean Hospital Corporation | 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 |
| CN108780473B (zh) * | 2016-01-21 | 2022-08-16 | 蛋白质动态解决方案有限公司 | 用于光谱数据分析的方法和系统 |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| CN109069496A (zh) * | 2016-04-29 | 2018-12-21 | 奥菲泽米有限公司 | 用于治疗葡糖脑苷脂酶相关疾病的arimoclomol |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| MX2019001421A (es) * | 2016-08-03 | 2019-09-04 | Univ South Florida | Composiciones de reelina para el tratamiento de trastornos neurologicos. |
| AU2018346104B2 (en) * | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20220010062A (ko) | 2017-10-03 | 2022-01-25 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20210261981A1 (en) * | 2017-10-23 | 2021-08-26 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| JP7522033B2 (ja) * | 2017-10-26 | 2024-07-24 | 武田薬品工業株式会社 | グルコセレブロシダーゼ及びイソファゴミンを含む製剤 |
| EP3953378A1 (en) * | 2019-04-10 | 2022-02-16 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| FI3953377T3 (fi) | 2019-04-10 | 2025-11-26 | Prevail Therapeutics Inc | Geeniterapioita lysosomaalisiin sairauksiin |
| MX2021015810A (es) | 2019-06-21 | 2022-08-15 | Broad Inst Inc | Agentes para revertir las proteinopatias toxicas. |
| 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 |
| CA3198606A1 (en) * | 2020-10-14 | 2022-04-21 | Denali Therapeutics Inc. | Methods for treating and monitoring frontotemporal dementia |
| CA3202568A1 (en) | 2020-11-19 | 2022-05-27 | Zevra Denmark 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 |
| EP4448763A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotide gba agonists |
| CN118382697A (zh) * | 2021-12-17 | 2024-07-23 | 豪夫迈·罗氏有限公司 | 能够增加葡糖脑苷脂酶表达的寡核苷酸 |
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| 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 |
| US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
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| DE69233745D1 (de) | 1991-12-02 | 2008-10-23 | Cambridge Antibody Tech | Herstellung von Autoantikörpern auf Phagenoberflächen ausgehend von Antikörpersegmentbibliotheken |
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| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
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| DK1143006T3 (da) | 1995-08-18 | 2008-07-14 | Morphosys Ip Gmbh | Vektorer/DNA-sekvenser fra humane kombinatoriske antistofbiblioteker |
| 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 |
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| DE60044997D1 (de) | 1999-07-02 | 2010-11-04 | Morphosys Ag | Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | 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 instrastrand base pairs |
| CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| US20020095135A1 (en) | 2000-06-19 | 2002-07-18 | David Meeker | 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 |
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| PL2154969T3 (pl) * | 2007-05-16 | 2016-04-29 | Brigham & Womens Hospital Inc | Leczenie synukleinopatii |
| EP2723384B1 (en) | 2011-06-22 | 2018-08-29 | The General Hospital Corporation | Treatment of proteinopathies |
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
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| TN2015000171A1 (fr) | 2016-10-03 |
| PH12015500879A1 (en) | 2015-06-29 |
| AU2013337354A1 (en) | 2015-05-21 |
| MX2015005722A (es) | 2016-01-12 |
| WO2014071282A1 (en) | 2014-05-08 |
| CL2015001157A1 (es) | 2015-10-16 |
| ZA201502618B (en) | 2016-01-27 |
| BR112015009746A2 (pt) | 2017-08-15 |
| JP2016503405A (ja) | 2016-02-04 |
| US20150284472A1 (en) | 2015-10-08 |
| SG11201502989XA (en) | 2015-05-28 |
| AR093355A1 (es) | 2015-06-03 |
| HK1214521A1 (zh) | 2016-07-29 |
| EP2914281A1 (en) | 2015-09-09 |
| CN104902923A (zh) | 2015-09-09 |
| TW201427695A (zh) | 2014-07-16 |
| CR20150216A (es) | 2015-05-29 |
| MA38144A1 (fr) | 2018-08-31 |
| IL238416A0 (en) | 2015-06-30 |
| KR20150079751A (ko) | 2015-07-08 |
| EA201590880A1 (ru) | 2015-09-30 |
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