TW201427695A - 用於治療蛋白質病變(proteinopathies)的組成物及方法 - Google Patents
用於治療蛋白質病變(proteinopathies)的組成物及方法 Download PDFInfo
- Publication number
- TW201427695A TW201427695A TW102139891A TW102139891A TW201427695A TW 201427695 A TW201427695 A TW 201427695A TW 102139891 A TW102139891 A TW 102139891A TW 102139891 A TW102139891 A TW 102139891A TW 201427695 A TW201427695 A TW 201427695A
- Authority
- TW
- Taiwan
- Prior art keywords
- protein
- gba1
- aav
- disease
- synuclein
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IG], 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
-
- 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 [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], 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)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychiatry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261722434P | 2012-11-05 | 2012-11-05 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW201427695A true TW201427695A (zh) | 2014-07-16 |
Family
ID=49578599
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW102139891A TW201427695A (zh) | 2012-11-05 | 2013-11-04 | 用於治療蛋白質病變(proteinopathies)的組成物及方法 |
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 |
|---|---|---|---|---|
| US8540985B2 (en) | 2008-06-26 | 2013-09-24 | 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 |
| CA2985235A1 (en) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
| WO2017015491A1 (en) * | 2015-07-21 | 2017-01-26 | Thomas Jefferson University | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
| CA3011719A1 (en) * | 2016-01-21 | 2017-07-27 | Protein Dynamic Solutions Llc | Method and system for spectral data analysis |
| EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
| KR20210059796A (ko) * | 2016-04-29 | 2021-05-25 | 오르파짐 에이/에스 | 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰 |
| 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 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| AU2018346104B2 (en) * | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| 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 |
| 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 |
| JP7664848B2 (ja) * | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス,インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2020257736A1 (en) | 2019-06-21 | 2020-12-24 | The Broad Institute, Inc. | Agents for reversing toxic proteinopathies |
| CN116390773A (zh) | 2020-08-06 | 2023-07-04 | 应用医学研究基金会 | 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒 |
| WO2022066911A1 (en) * | 2020-09-23 | 2022-03-31 | Seelos Therapeutics, Inc. | Compositions and methods for inhibiting alpha-synuclein aggregation |
| WO2022082178A2 (en) * | 2020-10-14 | 2022-04-21 | Denali Therapeutics Inc. | Methods for treating and monitoring frontotemporal dementia |
| KR20230128462A (ko) | 2020-11-19 | 2023-09-05 | 제브라 덴마크 에이/에스 | 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정 |
| 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 |
| 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 |
| 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 |
| 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 |
| US6172197B1 (en) | 1991-07-10 | 2001-01-09 | Medical Research Council | Methods for producing members of specific binding pairs |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
| ATE158021T1 (de) | 1990-08-29 | 1997-09-15 | Genpharm Int | Produktion und nützung nicht-menschliche transgentiere zur produktion heterologe antikörper |
| US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
| US5633425A (en) | 1990-08-29 | 1997-05-27 | 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 |
| CA2095633C (en) | 1990-12-03 | 2003-02-04 | Lisa J. Garrard | Enrichment method for variant proteins with altered binding properties |
| PT1024191E (pt) | 1991-12-02 | 2008-12-22 | Medical Res Council | Produção de auto-anticorpos a partir de reportórios de segmentos de anticorpo e exibidos em fagos |
| EP0705344B8 (en) | 1993-06-24 | 2006-05-10 | Advec Inc. | Adenovirus vectors for gene therapy |
| AU687117B2 (en) | 1993-10-25 | 1998-02-19 | Canji, Inc. | Recombinant adenoviral vector and methods of use |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| US7264963B1 (en) | 1995-08-18 | 2007-09-04 | Morphosys Ag | Protein(poly)peptide libraries |
| 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 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| 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 |
| 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 |
| 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 |
| WO2007150064A2 (en) * | 2006-06-23 | 2007-12-27 | Amicus Therapeutics, Inc. | METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE |
| EP2154969B1 (en) * | 2007-05-16 | 2015-11-18 | The Brigham and Women's Hospital, Inc. | Treatment of synucleinopathies |
| CA2840224C (en) | 2011-06-22 | 2019-08-13 | The General Hospital Corporation | Treatment of proteinopathies |
| 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 |
-
2013
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 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 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 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 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 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 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 MA MA38144A patent/MA38144A1/fr 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 |
|---|---|
| JP2016503405A (ja) | 2016-02-04 |
| HK1214521A1 (zh) | 2016-07-29 |
| MX2015005722A (es) | 2016-01-12 |
| CR20150216A (es) | 2015-05-29 |
| EA201590880A1 (ru) | 2015-09-30 |
| PH12015500879A1 (en) | 2015-06-29 |
| IL238416A0 (en) | 2015-06-30 |
| AU2013337354A1 (en) | 2015-05-21 |
| US20150284472A1 (en) | 2015-10-08 |
| CL2015001157A1 (es) | 2015-10-16 |
| SG11201502989XA (en) | 2015-05-28 |
| AR093355A1 (es) | 2015-06-03 |
| EP2914281A1 (en) | 2015-09-09 |
| BR112015009746A2 (pt) | 2017-08-15 |
| ZA201502618B (en) | 2016-01-27 |
| CA2889990A1 (en) | 2014-05-08 |
| KR20150079751A (ko) | 2015-07-08 |
| CN104902923A (zh) | 2015-09-09 |
| WO2014071282A1 (en) | 2014-05-08 |
| TN2015000171A1 (fr) | 2016-10-03 |
| MA38144A1 (fr) | 2018-08-31 |
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