AR093355A1 - Composiciones y metodos para tratar proteinopatias - Google Patents

Composiciones y metodos para tratar proteinopatias

Info

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
Application number
ARP130104033A
Other languages
English (en)
Spanish (es)
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49578599&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AR093355(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of AR093355A1 publication Critical patent/AR093355A1/es

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)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ARP130104033A 2012-11-05 2013-11-05 Composiciones y metodos para tratar proteinopatias AR093355A1 (es)

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 (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)

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EP2484371B1 (en) 2008-06-26 2014-12-17 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
WO2016179497A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. 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
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
SI3448382T1 (sl) 2016-04-29 2021-03-31 Orphazyme A/S, c/o COBIS A/S Arimoclomol za zdravljenje motenj, povezanih z glukocerebrozidazo
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
BR112020006633A2 (pt) * 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CN112553210B (zh) * 2017-10-03 2024-09-24 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078501A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
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
KR20220006527A (ko) * 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. 리소좀 장애에 대한 유전자 요법
US11999974B2 (en) 2019-04-10 2024-06-04 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
TW202214864A (zh) 2020-08-06 2022-04-16 應用醫學研究基金會 用於藉基因療法治療諸如阿茲海默症之Ｔａｕ蛋白病變的病毒顆粒
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
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
WO2022106614A1 (en) 2020-11-19 2022-05-27 Orphazyme 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
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
EP4448762A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotides capable of increasing glucocerebrosidase expression

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Also Published As

Publication number Publication date
US20150284472A1 (en) 2015-10-08
KR20150079751A (ko) 2015-07-08
WO2014071282A1 (en) 2014-05-08
MA38144A1 (fr) 2018-08-31
MX2015005722A (es) 2016-01-12
IL238416A0 (en) 2015-06-30
AU2013337354A1 (en) 2015-05-21
JP2016503405A (ja) 2016-02-04
EP2914281A1 (en) 2015-09-09
PH12015500879A1 (en) 2015-06-29
SG11201502989XA (en) 2015-05-28
CL2015001157A1 (es) 2015-10-16
ZA201502618B (en) 2016-01-27
CA2889990A1 (en) 2014-05-08
TW201427695A (zh) 2014-07-16
BR112015009746A2 (pt) 2017-08-15
HK1214521A1 (zh) 2016-07-29
EA201590880A1 (ru) 2015-09-30
CN104902923A (zh) 2015-09-09
TN2015000171A1 (fr) 2016-10-03
CR20150216A (es) 2015-05-29

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