BR112015009746A2 - Agente que aumenta atividade de glicocerebrosidase em mamíferos - Google Patents

Agente que aumenta atividade de glicocerebrosidase em mamíferos

Info

Publication number
BR112015009746A2
BR112015009746A2 BR112015009746A BR112015009746A BR112015009746A2 BR 112015009746 A2 BR112015009746 A2 BR 112015009746A2 BR 112015009746 A BR112015009746 A BR 112015009746A BR 112015009746 A BR112015009746 A BR 112015009746A BR 112015009746 A2 BR112015009746 A2 BR 112015009746A2
Authority
BR
Brazil
Prior art keywords
agent
increases
activity
mammals
mammal
Prior art date
Application number
BR112015009746A
Other languages
English (en)
Inventor
Pablo Sardi Sergio
Shihabuddin Lamya
Cheng Seng
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=BR112015009746(A2) "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 BR112015009746A2 publication Critical patent/BR112015009746A2/pt

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    • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (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)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

AGENTE QUE AUMENTA ATIVIDADE DE GLICOCEREBROSIDASE EM MAMÍFEROS. A presente invenção refere-se a processos para aperfeiçoamento de função neural em um mamífero com uma proteinopatia compreendendo administração de uma quantidade terapeuticamente efetiva de um agente que aumenta atividade de glicocerebrosidase no mamífero. Também são mostrados processos para redução de lipídeos tóxicos, redução de alfa-sinucleína, e/ou inibição de acumulação de agregados de proteínas em um mamífero com uma proteinopatia compreendendo administração de uma quantidade terapeuticamente efetiva de um agente que aumenta atividade de glicocerebrosidase.
BR112015009746A 2012-11-05 2013-11-04 Agente que aumenta atividade de glicocerebrosidase em mamíferos BR112015009746A2 (pt)

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
BR112015009746A2 true BR112015009746A2 (pt) 2017-08-15

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015009746A BR112015009746A2 (pt) 2012-11-05 2013-11-04 Agente que aumenta atividade de glicocerebrosidase em mamíferos

Country Status (22)

Country Link
US (1) US20150284472A1 (pt)
EP (1) EP2914281A1 (pt)
JP (1) JP2016503405A (pt)
KR (1) KR20150079751A (pt)
CN (1) CN104902923A (pt)
AR (1) AR093355A1 (pt)
AU (1) AU2013337354A1 (pt)
BR (1) BR112015009746A2 (pt)
CA (1) CA2889990A1 (pt)
CL (1) CL2015001157A1 (pt)
CR (1) CR20150216A (pt)
EA (1) EA201590880A1 (pt)
HK (1) HK1214521A1 (pt)
IL (1) IL238416A0 (pt)
MA (1) MA38144A1 (pt)
MX (1) MX2015005722A (pt)
PH (1) PH12015500879A1 (pt)
SG (1) SG11201502989XA (pt)
TN (1) TN2015000171A1 (pt)
TW (1) TW201427695A (pt)
WO (1) WO2014071282A1 (pt)
ZA (1) ZA201502618B (pt)

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EP3193840B1 (en) 2014-09-15 2021-05-19 Orphazyme A/S Arimoclomol formulation
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited 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
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EP3765855A1 (en) * 2018-03-14 2021-01-20 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
CN114174324A (zh) 2019-04-10 2022-03-11 普利维尔治疗公司 用于溶酶体病症的基因疗法
EP3953378A1 (en) * 2019-04-10 2022-02-16 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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JP2023536080A (ja) 2020-08-06 2023-08-23 ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ 遺伝子治療によるアルツハイマー病などのタウオパチーの治療に使用するためのウイルス粒子
CA3202568A1 (en) 2020-11-19 2022-05-27 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
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Also Published As

Publication number Publication date
TW201427695A (zh) 2014-07-16
MA38144A1 (fr) 2018-08-31
CR20150216A (es) 2015-05-29
AU2013337354A1 (en) 2015-05-21
EP2914281A1 (en) 2015-09-09
CL2015001157A1 (es) 2015-10-16
TN2015000171A1 (fr) 2016-10-03
KR20150079751A (ko) 2015-07-08
CA2889990A1 (en) 2014-05-08
WO2014071282A1 (en) 2014-05-08
MX2015005722A (es) 2016-01-12
IL238416A0 (en) 2015-06-30
CN104902923A (zh) 2015-09-09
JP2016503405A (ja) 2016-02-04
ZA201502618B (en) 2016-01-27
HK1214521A1 (zh) 2016-07-29
US20150284472A1 (en) 2015-10-08
AR093355A1 (es) 2015-06-03
PH12015500879A1 (en) 2015-06-29
EA201590880A1 (ru) 2015-09-30
SG11201502989XA (en) 2015-05-28

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