AR093355A1 - Composiciones y metodos para tratar proteinopatias - Google Patents
Composiciones y metodos para tratar proteinopatiasInfo
- Publication number
- AR093355A1 AR093355A1 ARP130104033A ARP130104033A AR093355A1 AR 093355 A1 AR093355 A1 AR 093355A1 AR P130104033 A ARP130104033 A AR P130104033A AR P130104033 A ARP130104033 A AR P130104033A AR 093355 A1 AR093355 A1 AR 093355A1
- Authority
- AR
- Argentina
- Prior art keywords
- agent
- reducing
- mammal
- methods
- proteinopathy
- 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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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)
Abstract
Métodos para mejorar la función neural en un mamífero con una proteinopatía, que comprende administrar una cantidad terapéuticamente eficaz de un agente que aumenta la actividad glucocerebrosidasa en el mamífero. También se describen métodos para reducir los lípidos tóxicos, reducir la a-sinucleína, y/o inhibir la acumulación de agregados de proteínas en un mamífero con una proteinopatía, que comprende administrar una cantidad terapéuticamente eficaz de un agente que aumenta la actividad glucocerebrosidasa. Reivindicación 5: Un método para reducir los lípidos tóxicos, reducir la a-sinucleína, reducir tau o inhibir la acumulación de agregados de proteínas en un mamífero con una proteinopatía, que comprende administrar una cantidad terapéuticamente eficaz de un agente que aumenta la actividad glucocerebrosidasa. Reivindicación 24: El método de una cualquiera de las reivindicaciones 1 - 23, en donde el agente comprende una molécula pequeña, un anticuerpo, una molécula de ácido nucleico, o un polipéptido. Reivindicación 25: El método de la reivindicación 24, en donde el agente es un ácido nucleico que codifica un gen de GBA1 o equivalente del mismo. Reivindicación 26: El método de la reivindicación 24, en donde el agente es un polipéptido GBA1 o equivalente del mismo. Reivindicación 27: El método de la reivindicación 24, en donde el agente es un anticuerpo que se une específicamente a GBA1. Reivindicación 30: El método de la reivindicación 24, en donde el agente es un virus. Reivindicación 34: El método de una cualquiera de las reivindicaciones 30 - 33, en donde el virus es un virus adeno-asociado (AAV).
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261722434P | 2012-11-05 | 2012-11-05 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR093355A1 true AR093355A1 (es) | 2015-06-03 |
Family
ID=49578599
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP130104033A AR093355A1 (es) | 2012-11-05 | 2013-11-05 | Composiciones y metodos para tratar proteinopatias |
Country Status (22)
Country | Link |
---|---|
US (1) | US20150284472A1 (es) |
EP (1) | EP2914281A1 (es) |
JP (1) | JP2016503405A (es) |
KR (1) | KR20150079751A (es) |
CN (1) | CN104902923A (es) |
AR (1) | AR093355A1 (es) |
AU (1) | AU2013337354A1 (es) |
BR (1) | BR112015009746A2 (es) |
CA (1) | CA2889990A1 (es) |
CL (1) | CL2015001157A1 (es) |
CR (1) | CR20150216A (es) |
EA (1) | EA201590880A1 (es) |
HK (1) | HK1214521A1 (es) |
IL (1) | IL238416A0 (es) |
MA (1) | MA38144A1 (es) |
MX (1) | MX2015005722A (es) |
PH (1) | PH12015500879A1 (es) |
SG (1) | SG11201502989XA (es) |
TN (1) | TN2015000171A1 (es) |
TW (1) | TW201427695A (es) |
WO (1) | WO2014071282A1 (es) |
ZA (1) | ZA201502618B (es) |
Families Citing this family (23)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3004867C (en) | 2008-06-26 | 2020-09-15 | Orphazyme Aps | Use of hsp70 as a regulator of enzymatic activity |
WO2012072082A1 (en) | 2010-11-30 | 2012-06-07 | Orphazyme Aps | Methods for increasing intracellular activity of hsp70 |
PL3193840T3 (pl) | 2014-09-15 | 2021-12-06 | Orphazyme A/S | Preparat arimoklomolu |
EP3292206B8 (en) * | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | 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 |
WO2017178029A1 (en) | 2016-04-13 | 2017-10-19 | Orphazyme Aps | Heat shock proteins and cholesterol homeostasis |
PT3448382T (pt) * | 2016-04-29 | 2020-11-20 | Orphazyme As C/O Cobis As | Arimoclomol para o tratamento de distúrbios associados à glucocerebrosidase |
EP3264092A1 (en) * | 2016-07-01 | 2018-01-03 | Centogene AG | Use of lyso-gb1 as druggable target |
EP3506927A4 (en) * | 2016-08-03 | 2020-05-06 | University of South Florida | REELINE COMPOSITIONS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS |
CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
AU2018346105C1 (en) | 2017-10-03 | 2023-10-12 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN111542549A (zh) * | 2017-10-03 | 2020-08-14 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
AU2018354195A1 (en) * | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
JP2021501135A (ja) * | 2017-10-26 | 2021-01-14 | シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド | 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 |
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 |
EP3953378A1 (en) * | 2019-04-10 | 2022-02-16 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
AU2020273182A1 (en) | 2019-04-10 | 2021-12-02 | 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 |
MX2023005954A (es) | 2020-11-19 | 2023-09-04 | Zevra Denmark As | Procesos para preparar citrato de arimoclomol e intermediarios del mismo. |
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 |
WO2023111336A1 (en) * | 2021-12-17 | 2023-06-22 | F. Hoffmann-La Roche Ag | Oligonucleotide gba agonists |
WO2023111335A1 (en) * | 2021-12-17 | 2023-06-22 | F. Hoffmann-La Roche Ag | Oligonucleotides capable of increasing glucocerebrosidase expression |
Family Cites Families (33)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
US5661016A (en) | 1990-08-29 | 1997-08-26 | Genpharm International Inc. | Transgenic non-human animals capable of producing heterologous antibodies of various isotypes |
KR100272077B1 (ko) | 1990-08-29 | 2000-11-15 | 젠팜인터내셔날,인코포레이티드 | 이종 항체를 생산할 수 있는 전이유전자를 가진 인간이외의 동물 |
US5625126A (en) | 1990-08-29 | 1997-04-29 | Genpharm International, Inc. | Transgenic non-human animals for producing heterologous antibodies |
US5633425A (en) | 1990-08-29 | 1997-05-27 | Genpharm International, Inc. | Transgenic non-human animals capable of producing heterologous antibodies |
US5545806A (en) | 1990-08-29 | 1996-08-13 | Genpharm International, Inc. | Ransgenic non-human animals for producing heterologous antibodies |
CA2405246A1 (en) | 1990-12-03 | 1992-06-11 | Genentech, Inc. | Enrichment method for variant proteins with alterred binding properties |
ES2341666T3 (es) | 1991-12-02 | 2010-06-24 | Medimmune Limited | Produccion de autoanticuerpos de repertorios de segmentos de anticue rpos expresados en la superficie de fagos. |
EP0705344B8 (en) | 1993-06-24 | 2006-05-10 | Advec Inc. | Adenovirus vectors for gene therapy |
BR9407956A (pt) | 1993-10-25 | 1996-11-26 | Canji Inc | Composiçao farmacêutica vetor de express o de adenovirus recombinante e kit par reduzir a proliferaçao de células tumorais |
US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
US6706484B1 (en) | 1995-08-18 | 2004-03-16 | Morphosys Ag | Protein/(poly)peptide libraries |
JP4436457B2 (ja) | 1995-08-18 | 2010-03-24 | モルフォシス アイピー ゲーエムベーハー | 蛋白質/(ポリ)ペプチドライブラリー |
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 |
CA2339889C (en) | 1999-07-02 | 2012-01-31 | Morphosys Ag | Identification of specific binding partners binding to (poly)peptides encoded by genomic dna fragments or ests |
JP4827353B2 (ja) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大 |
DE60117550T2 (de) | 2000-06-01 | 2006-12-07 | University Of North Carolina At Chapel Hill | Doppelsträngige parvovirus-vektoren |
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 |
US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
US7829579B2 (en) * | 2006-06-23 | 2010-11-09 | Amicus Therapeutics, Inc. | Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase |
CN101754682B (zh) * | 2007-05-16 | 2014-11-12 | 布里格姆妇女医院 | 突触核蛋白病的治疗 |
EP2723384B1 (en) | 2011-06-22 | 2018-08-29 | 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 CN CN201380069582.2A patent/CN104902923A/zh active Pending
- 2013-11-04 SG SG11201502989XA patent/SG11201502989XA/en unknown
- 2013-11-04 AU AU2013337354A patent/AU2013337354A1/en not_active Abandoned
- 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 EP EP13789690.8A patent/EP2914281A1/en not_active Withdrawn
- 2013-11-04 MA MA38144A patent/MA38144A1/fr unknown
- 2013-11-04 TW TW102139891A patent/TW201427695A/zh unknown
- 2013-11-04 EA EA201590880A patent/EA201590880A1/ru unknown
- 2013-11-04 KR KR1020157013541A patent/KR20150079751A/ko not_active Application Discontinuation
- 2013-11-04 WO PCT/US2013/068242 patent/WO2014071282A1/en active Application Filing
- 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 MX MX2015005722A patent/MX2015005722A/es 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
Also Published As
Publication number | Publication date |
---|---|
IL238416A0 (en) | 2015-06-30 |
CR20150216A (es) | 2015-05-29 |
CA2889990A1 (en) | 2014-05-08 |
HK1214521A1 (zh) | 2016-07-29 |
CN104902923A (zh) | 2015-09-09 |
CL2015001157A1 (es) | 2015-10-16 |
AU2013337354A1 (en) | 2015-05-21 |
KR20150079751A (ko) | 2015-07-08 |
BR112015009746A2 (pt) | 2017-08-15 |
EA201590880A1 (ru) | 2015-09-30 |
PH12015500879A1 (en) | 2015-06-29 |
MX2015005722A (es) | 2016-01-12 |
SG11201502989XA (en) | 2015-05-28 |
US20150284472A1 (en) | 2015-10-08 |
ZA201502618B (en) | 2016-01-27 |
WO2014071282A1 (en) | 2014-05-08 |
EP2914281A1 (en) | 2015-09-09 |
TW201427695A (zh) | 2014-07-16 |
TN2015000171A1 (fr) | 2016-10-03 |
MA38144A1 (fr) | 2018-08-31 |
JP2016503405A (ja) | 2016-02-04 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
AR093355A1 (es) | Composiciones y metodos para tratar proteinopatias | |
EA201591992A1 (ru) | Эффективная доставка больших генов посредством двойных aav векторов | |
BR112016012968A2 (pt) | Métodos e composições para o tratamento de condições associadas com o envelhecimento | |
CO2017006580A2 (es) | Anticuerpos humanos para hemaglutinina de influenza | |
BR112017013599A2 (pt) | composição farmacêutica, e, método para prevenir, tratar ou melhorar um ou mais sintomas de um tumor maligno associado à mutação de kras. | |
CL2018001522A1 (es) | Focalización de péptidos para dirigir virus adenoasociados (aavs) | |
BR112018068189A2 (pt) | proteínas de ligação induzíveis e métodos de uso | |
BR112018008766A2 (pt) | variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos | |
BR112017002781A2 (pt) | cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia | |
EA201500802A1 (ru) | Способы профилактики и/или лечения кардиомиопатии и устранения симптомов кардиомиопатии, фармацевтическая композиция, векторы | |
BR112014028666A2 (pt) | métodos e composições para o tratamento de depósitos amiloides | |
CL2016000502A1 (es) | Vectores mejorados del factor viii del virus adeno-asociado de menos de 5,0 kb de longitud, que producen un polipéptido del factor viii funcionalmente activo para el tratamiento de la hemofilia a. | |
EA201790378A1 (ru) | Заключенное в липосому аффинное лекарственное средство | |
PE20150163A1 (es) | Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav | |
MY194175A (en) | Variant rnai | |
EA201491644A1 (ru) | Фармацевтические композиции | |
CL2015000352A1 (es) | Métodos de tratamiento de una tauopatía | |
CL2013003214A1 (es) | Formulacion estable que comprende al menos un anticuerpo monoclonal que se une especificamente a pcsk9, un tampon, un surfactante y al menos un estabilizador; y su uso para tratar o prevenir enfermedades relacionadas con el colesterol. | |
EA201791936A8 (ru) | Рекомбинантные конструкции glut1 аденоассоциированного вирусного вектора и способы восстановления экспрессии glut1 на их основе | |
EA201690377A1 (ru) | Антитела к ингибитору активатора плазминогена 1 (pai-1) и пути их применения | |
UY35703A (es) | Anticuerpos contra el inhibidor-1 del activador de plasminógeno (pai-1) y usos de los mismos | |
DOP2017000248A (es) | PROTEÍNA DE UNIÓN A RGMa Y SU USO | |
EA201592203A1 (ru) | Способы лечения таупатии | |
BR112017005235A2 (pt) | vetores virais adeno-associados para o tratamento de glaucoma de miocilina (myoc) | |
AR091155A1 (es) | Composicion farmaceutica acuosa que contiene un agente terapeutico biologico y guanidina o un derivado de guanidina y una inyeccion que incluye la composicion |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
FB | Suspension of granting procedure |