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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
-
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
-
- 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
-
- 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)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychiatry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 (1)
| Publication Number | Publication Date |
|---|---|
| CA2889990A1 true CA2889990A1 (en) | 2014-05-08 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2889990A Abandoned CA2889990A1 (en) | 2012-11-05 | 2013-11-04 | Compositions and methods for treating proteinopathies |
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) |
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 |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FI3578195T3 (fi) | 2008-06-26 | 2023-11-08 | Zevra Denmark As | Hsp70:n käyttö entsyymitoiminnan säätelijänä |
| RU2013125923A (ru) | 2010-11-30 | 2015-01-10 | Орфазиме Апс | СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70 |
| JP6678676B2 (ja) | 2014-09-15 | 2020-04-08 | オーファザイム エー／エス | アリモクロモル製剤 |
| EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
| US10874749B2 (en) * | 2015-07-21 | 2020-12-29 | Thomas Jefferson University | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| EP3405898A1 (en) * | 2016-01-21 | 2018-11-28 | Protein Dynamic Solutions LLC | Method and system for spectral data analysis |
| WO2017178029A1 (en) | 2016-04-13 | 2017-10-19 | Orphazyme Aps | Heat shock proteins and cholesterol homeostasis |
| AU2017255959B2 (en) * | 2016-04-29 | 2020-10-15 | Zevra Denmark A/S | Arimoclomol for treating glucocerebrosidase associated disorders |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| CA3032697A1 (en) * | 2016-08-03 | 2018-02-08 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| CA3078371A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal 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 |
| AU2018346105C1 (en) | 2017-10-03 | 2023-10-12 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| KR20200075865A (ko) * | 2017-10-23 | 2020-06-26 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환에 대한 유전자 요법 |
| CN111278439A (zh) * | 2017-10-26 | 2020-06-12 | 夏尔人类遗传性治疗公司 | 包含葡萄糖脑苷脂酶和异烟肼的调配物 |
| HRP20251506T1 (hr) | 2019-04-10 | 2026-01-02 | Prevail Therapeutics, Inc. | Genska terapija za lizosomske poremećaje |
| MX2021012184A (es) * | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | AGENTS TO REVERSE TOXIC PROTEINOPATHIES |
| US20230277687A1 (en) | 2020-08-06 | 2023-09-07 | Fundacion Para La Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| 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 |
| 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 |
| EP4448763A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotide gba agonists |
| EP4448762A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotides capable of increasing glucocerebrosidase expression |
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| US4816567A (en) | 1983-04-08 | 1989-03-28 | Genentech, Inc. | Recombinant immunoglobin preparations |
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| 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 |
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| DE69127627T2 (de) | 1990-08-29 | 1998-02-19 | Genpharm Int | Produktion und Nützung nicht-menschliche transgentiere zur Produktion heterologe Antikörper |
| ATE164395T1 (de) | 1990-12-03 | 1998-04-15 | Genentech Inc | Verfahren zur anreicherung von proteinvarianten mit geänderten bindungseigenschaften |
| ATE408012T1 (de) | 1991-12-02 | 2008-09-15 | Medical Res Council | Herstellung von autoantikörpern auf phagenoberflächen ausgehend von antikörpersegmentbibliotheken |
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| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | 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. |
| ES2505700T3 (es) | 2000-06-01 | 2014-10-10 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados |
| 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 |
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| PL2154969T3 (pl) * | 2007-05-16 | 2016-04-29 | Brigham & Womens Hospital Inc | Leczenie synukleinopatii |
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| JP2015512913A (ja) | 2012-03-28 | 2015-04-30 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | グルコセレブロシダーゼ活性剤として有用なサリチル酸誘導体 |
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2013
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- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en not_active Ceased
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 BR BR112015009746A patent/BR112015009746A2/pt not_active IP Right Cessation
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 HK HK16102536.9A patent/HK1214521A1/zh unknown
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-04 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 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
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 |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20150079751A (ko) | 2015-07-08 |
| CN104902923A (zh) | 2015-09-09 |
| AU2013337354A1 (en) | 2015-05-21 |
| CR20150216A (es) | 2015-05-29 |
| TW201427695A (zh) | 2014-07-16 |
| HK1214521A1 (zh) | 2016-07-29 |
| US20150284472A1 (en) | 2015-10-08 |
| EA201590880A1 (ru) | 2015-09-30 |
| ZA201502618B (en) | 2016-01-27 |
| MA38144A1 (fr) | 2018-08-31 |
| EP2914281A1 (en) | 2015-09-09 |
| CL2015001157A1 (es) | 2015-10-16 |
| IL238416A0 (en) | 2015-06-30 |
| MX2015005722A (es) | 2016-01-12 |
| SG11201502989XA (en) | 2015-05-28 |
| TN2015000171A1 (fr) | 2016-10-03 |
| WO2014071282A1 (en) | 2014-05-08 |
| PH12015500879A1 (en) | 2015-06-29 |
| JP2016503405A (ja) | 2016-02-04 |
| AR093355A1 (es) | 2015-06-03 |
| BR112015009746A2 (pt) | 2017-08-15 |
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