EA201590880A1 - Композиции и способы лечения протеинопатий - Google Patents
Композиции и способы лечения протеинопатийInfo
- Publication number
- EA201590880A1 EA201590880A1 EA201590880A EA201590880A EA201590880A1 EA 201590880 A1 EA201590880 A1 EA 201590880A1 EA 201590880 A EA201590880 A EA 201590880A EA 201590880 A EA201590880 A EA 201590880A EA 201590880 A1 EA201590880 A1 EA 201590880A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- proteinopathy
- methods
- mammal
- compositions
- treatment
- Prior art date
Links
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
-
- 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)
- 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)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Plant Pathology (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Данное изобретение относится к способам улучшения функции нервной системы у млекопитающего с протеинопатией, включающим введение терапевтически эффективного количества средства, которое повышает глюкоцереброзидазную активность у млекопитающего. Также описаны способы уменьшения токсических липидов, уменьшения α-синуклеина и/или ингибирования накопления белковых агрегатов у млекопитающего с протеинопатией, включающие введение терапевтически эффективного количества средства, которое повышает глюкоцереброзидазную активность.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261722434P | 2012-11-05 | 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 |
---|---|
EA201590880A1 true EA201590880A1 (ru) | 2015-09-30 |
Family
ID=49578599
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201590880A EA201590880A1 (ru) | 2012-11-05 | 2013-11-04 | Композиции и способы лечения протеинопатий |
Country Status (22)
Country | Link |
---|---|
US (1) | US20150284472A1 (ru) |
EP (1) | EP2914281A1 (ru) |
JP (1) | JP2016503405A (ru) |
KR (1) | KR20150079751A (ru) |
CN (1) | CN104902923A (ru) |
AR (1) | AR093355A1 (ru) |
AU (1) | AU2013337354A1 (ru) |
BR (1) | BR112015009746A2 (ru) |
CA (1) | CA2889990A1 (ru) |
CL (1) | CL2015001157A1 (ru) |
CR (1) | CR20150216A (ru) |
EA (1) | EA201590880A1 (ru) |
HK (1) | HK1214521A1 (ru) |
IL (1) | IL238416A0 (ru) |
MA (1) | MA38144A1 (ru) |
MX (1) | MX2015005722A (ru) |
PH (1) | PH12015500879A1 (ru) |
SG (1) | SG11201502989XA (ru) |
TN (1) | TN2015000171A1 (ru) |
TW (1) | TW201427695A (ru) |
WO (1) | WO2014071282A1 (ru) |
ZA (1) | ZA201502618B (ru) |
Cited By (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RU2750154C2 (ru) * | 2016-04-29 | 2021-06-22 | Орфазим А/С | Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений |
Families Citing this family (22)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
PL2659904T3 (pl) | 2008-06-26 | 2016-01-29 | Orphazyme Aps | Zastosowanie Hsp70 jako regulatora aktywności enzymatycznej |
BR112013013143A2 (pt) | 2010-11-30 | 2016-08-23 | Orphazyme Aps | composto, e, método de tratamento de uma doença de armazenagem lisossômica |
KR20240028540A (ko) | 2014-09-15 | 2024-03-05 | 제브라 덴마크 에이/에스 | 아리모클로몰 제제 |
CA2985235A1 (en) * | 2015-05-07 | 2016-11-10 | Shire Human Genetic Therapies, Inc. | Glucocerebrosidase gene therapy for parkinson's disease |
CA3030261A1 (en) * | 2015-07-21 | 2017-01-26 | Thomas Jefferson University | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
EP3442530A1 (en) | 2016-04-13 | 2019-02-20 | Orphazyme A/S | Heat shock proteins and cholesterol homeostasis |
EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
CN109715194B (zh) * | 2016-08-03 | 2024-06-11 | 南佛罗里达大学 | 用于治疗神经障碍的颤蛋白组合物 |
US11993790B2 (en) | 2017-10-03 | 2024-05-28 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
WO2019070891A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISORDERS |
AU2018346104B2 (en) * | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN111819281A (zh) | 2017-10-23 | 2020-10-23 | 普利维尔治疗公司 | 用于神经变性疾病的基因疗法 |
CA3078463A1 (en) * | 2017-10-26 | 2019-05-02 | Shire Human Genetic Therapies, Inc. | Formulations comprising glucocerebrosidase and isofagomine |
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 |
BR112021020183A2 (pt) | 2019-04-10 | 2022-03-03 | Prevail Therapeutics Inc | Terapias gênicas para distúrbios lisossomais |
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 |
AR123168A1 (es) | 2020-08-06 | 2022-11-02 | Fundacion Para La Investig Medica Aplicada | Partículas virales para uso en el tratamiento de tauopatías tales como enfermedades de alzheimer mediante terapia génica |
US20230416224A1 (en) | 2020-11-19 | 2023-12-28 | 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 |
CN118489009A (zh) * | 2021-12-17 | 2024-08-13 | 豪夫迈·罗氏有限公司 | 寡核苷酸gba激动剂 |
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 |
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 |
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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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US5633425A (en) | 1990-08-29 | 1997-05-27 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
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WO1993011236A1 (en) | 1991-12-02 | 1993-06-10 | Medical Research Council | Production of anti-self antibodies from antibody segment repertoires and displayed on phage |
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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US7264963B1 (en) | 1995-08-18 | 2007-09-04 | Morphosys Ag | Protein(poly)peptide libraries |
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 |
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ES2353268T3 (es) | 1999-07-02 | 2011-02-28 | Morphosys Ag | Generacion de elementos de union especifica que se unen a (poli)peptidos codificados por fragmentos de adn genomico o est. |
PT1204739E (pt) | 1999-08-09 | 2008-11-17 | Targeted Genetics Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
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 |
JP5291093B2 (ja) * | 2007-05-16 | 2013-09-18 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド | シヌクレイノパチーの治療 |
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 US US14/440,818 patent/US20150284472A1/en not_active Abandoned
- 2013-11-04 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 JP JP2015540845A patent/JP2016503405A/ja not_active Abandoned
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 MX MX2015005722A patent/MX2015005722A/es unknown
- 2013-11-04 CA CA2889990A patent/CA2889990A1/en not_active Abandoned
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 2013-11-04 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 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 WO PCT/US2013/068242 patent/WO2014071282A1/en active Application Filing
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Application Discontinuation
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh 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
-
2016
- 2016-03-07 HK HK16102536.9A patent/HK1214521A1/zh unknown
Cited By (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
RU2750154C2 (ru) * | 2016-04-29 | 2021-06-22 | Орфазим А/С | Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений |
Also Published As
Publication number | Publication date |
---|---|
TW201427695A (zh) | 2014-07-16 |
SG11201502989XA (en) | 2015-05-28 |
CL2015001157A1 (es) | 2015-10-16 |
TN2015000171A1 (fr) | 2016-10-03 |
EP2914281A1 (en) | 2015-09-09 |
ZA201502618B (en) | 2016-01-27 |
WO2014071282A1 (en) | 2014-05-08 |
MX2015005722A (es) | 2016-01-12 |
AR093355A1 (es) | 2015-06-03 |
BR112015009746A2 (pt) | 2017-08-15 |
CA2889990A1 (en) | 2014-05-08 |
CR20150216A (es) | 2015-05-29 |
CN104902923A (zh) | 2015-09-09 |
MA38144A1 (fr) | 2018-08-31 |
HK1214521A1 (zh) | 2016-07-29 |
US20150284472A1 (en) | 2015-10-08 |
IL238416A0 (en) | 2015-06-30 |
KR20150079751A (ko) | 2015-07-08 |
AU2013337354A1 (en) | 2015-05-21 |
JP2016503405A (ja) | 2016-02-04 |
PH12015500879A1 (en) | 2015-06-29 |
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