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

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use 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)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (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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KR20250069686A (ko) 2014-09-15 2025-05-19 제브라 덴마크 에이/에스 아리모클로몰 제제
AU2016256895B2 (en) * 2015-05-07 2022-05-26 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
CN108780473B (zh) * 2016-01-21 2022-08-16 蛋白质动态解决方案有限公司 用于光谱数据分析的方法和系统
EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
ES2831764T3 (es) * 2016-04-29 2021-06-09 Orphazyme As Arimoclomol para el tratamiento de trastornos asociados a glucocerebrosidasa
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
KR102523237B1 (ko) * 2016-08-03 2023-04-18 유니버시티 오브 사우스 플로리다 신경 장애의 치료를 위한 리일린 조성물
BR112020006633A2 (pt) * 2017-10-03 2020-10-06 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7361037B2 (ja) * 2017-10-03 2023-10-13 プリベイル セラピューティクス,インコーポレーテッド ライソゾーム病の遺伝子治療
CA3083582A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
US20200316178A1 (en) * 2017-10-26 2020-10-08 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
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
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
WO2020257736A1 (en) 2019-06-21 2020-12-24 The Broad Institute, Inc. Agents for reversing toxic proteinopathies
CA3181024A1 (en) 2020-08-06 2022-02-10 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
CN116420078A (zh) * 2020-10-14 2023-07-11 戴纳立制药公司 用于治疗和监测额颞痴呆的方法
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
EP4448762A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotides capable of increasing glucocerebrosidase expression
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

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Also Published As

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

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