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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- 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
- A61P25/28—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)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (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
ID=49578599
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 (OSRAM) |
| EP (1) | EP2914281A1 (OSRAM) |
| JP (1) | JP2016503405A (OSRAM) |
| KR (1) | KR20150079751A (OSRAM) |
| CN (1) | CN104902923A (OSRAM) |
| AR (1) | AR093355A1 (OSRAM) |
| AU (1) | AU2013337354A1 (OSRAM) |
| BR (1) | BR112015009746A2 (OSRAM) |
| CA (1) | CA2889990A1 (OSRAM) |
| CL (1) | CL2015001157A1 (OSRAM) |
| CR (1) | CR20150216A (OSRAM) |
| EA (1) | EA201590880A1 (OSRAM) |
| HK (1) | HK1214521A1 (OSRAM) |
| IL (1) | IL238416A0 (OSRAM) |
| MA (1) | MA38144A1 (OSRAM) |
| MX (1) | MX2015005722A (OSRAM) |
| PH (1) | PH12015500879A1 (OSRAM) |
| SG (1) | SG11201502989XA (OSRAM) |
| TN (1) | TN2015000171A1 (OSRAM) |
| TW (1) | TW201427695A (OSRAM) |
| WO (1) | WO2014071282A1 (OSRAM) |
| ZA (1) | ZA201502618B (OSRAM) |
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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| Publication number | Priority date | Publication date | Assignee | Title |
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| CA2728363C (en) | 2008-06-26 | 2019-02-19 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
| PL2646044T3 (pl) | 2010-11-30 | 2020-03-31 | Orphazyme A/S | Sposoby zwiększenia aktywności wewnątrzkomórkowej 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 |
| AU2016256895B2 (en) * | 2015-05-07 | 2022-05-26 | Takeda Pharmaceutical Company Limited | 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 |
| US10898476B2 (en) | 2016-04-13 | 2021-01-26 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| BR112018070653A2 (pt) * | 2016-04-29 | 2019-02-05 | Orphazyme As | ingrediente farmacêutico ativo, e, composição |
| EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
| WO2018027037A2 (en) * | 2016-08-03 | 2018-02-08 | University Of South Florida | Reelin compositions for treatment of neurological disorders |
| US11802294B2 (en) * | 2017-10-03 | 2023-10-31 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020008033A2 (pt) * | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
| EA202091032A1 (ru) * | 2017-10-26 | 2020-07-17 | Шайр Хьюман Дженетик Терапиз, Инк. | Составы, содержащие глюкоцереброзидазу и изофагомин |
| DK3953377T3 (da) | 2019-04-10 | 2025-12-01 | Prevail Therapeutics Inc | Genterapier til lysosomale lidelser |
| WO2020210713A1 (en) * | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| EP3986412A4 (en) | 2019-06-21 | 2023-11-15 | The Broad Institute, Inc. | AGENTS TO REVERSE TOXIC PROTEINOPATHIES |
| CA3181024A1 (en) | 2020-08-06 | 2022-02-10 | 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 |
| AU2021380947C1 (en) | 2020-11-19 | 2025-02-20 | 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 |
| EP4448762A1 (en) * | 2021-12-17 | 2024-10-23 | F. Hoffmann-La Roche AG | Oligonucleotides capable of increasing glucocerebrosidase expression |
| 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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| 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 |
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| 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 |
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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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| JP3532566B2 (ja) | 1993-06-24 | 2004-05-31 | エル． グラハム，フランク | 遺伝子治療のためのアデノウイルスベクター |
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| WO2001002588A2 (en) | 1999-07-02 | 2001-01-11 | Morphosys Ag | Generation of specific binding partners binding to (poly)peptides encoded by genomic dna fragments or ests |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| 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 |
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-
2013
- 2013-11-04 BR BR112015009746A patent/BR112015009746A2/pt not_active IP Right Cessation
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Withdrawn
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 HK HK16102536.9A patent/HK1214521A1/zh 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 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 US US14/440,818 patent/US20150284472A1/en 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 |
|---|---|
| MX2015005722A (es) | 2016-01-12 |
| IL238416A0 (en) | 2015-06-30 |
| BR112015009746A2 (pt) | 2017-08-15 |
| AR093355A1 (es) | 2015-06-03 |
| EA201590880A1 (ru) | 2015-09-30 |
| KR20150079751A (ko) | 2015-07-08 |
| ZA201502618B (en) | 2016-01-27 |
| EP2914281A1 (en) | 2015-09-09 |
| CL2015001157A1 (es) | 2015-10-16 |
| MA38144A1 (fr) | 2018-08-31 |
| WO2014071282A1 (en) | 2014-05-08 |
| JP2016503405A (ja) | 2016-02-04 |
| US20150284472A1 (en) | 2015-10-08 |
| CR20150216A (es) | 2015-05-29 |
| HK1214521A1 (zh) | 2016-07-29 |
| TW201427695A (zh) | 2014-07-16 |
| AU2013337354A1 (en) | 2015-05-21 |
| CN104902923A (zh) | 2015-09-09 |
| PH12015500879A1 (en) | 2015-06-29 |
| SG11201502989XA (en) | 2015-05-28 |
| TN2015000171A1 (fr) | 2016-10-03 |
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