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EA201590880A1 - Композиции и способы лечения протеинопатий - Google Patents

Композиции и способы лечения протеинопатий

Info

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
Application number
EA201590880A
Other languages
English (en)
Inventor
Серджио Пабло Сарди
Лэмайа Шихабуддин
Сэн Чэн
Original Assignee
Джензим Корпорейшн
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=EA201590880(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Джензим Корпорейшн filed Critical Джензим Корпорейшн
Publication of EA201590880A1 publication Critical patent/EA201590880A1/ru

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)
  • 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.
EA201590880A 2012-11-05 2013-11-04 Композиции и способы лечения протеинопатий EA201590880A1 (ru)

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)

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RU2750154C2 (ru) * 2016-04-29 2021-06-22 Орфазим А/С Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений

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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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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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