TN2015000171A1 - Compositions and methods for treating proteinopathies - Google Patents

Compositions and methods for treating proteinopathies

Info

Publication number
TN2015000171A1
TN2015000171A1 TNP2015000171A TN2015000171A TN2015000171A1 TN 2015000171 A1 TN2015000171 A1 TN 2015000171A1 TN P2015000171 A TNP2015000171 A TN P2015000171A TN 2015000171 A TN2015000171 A TN 2015000171A TN 2015000171 A1 TN2015000171 A1 TN 2015000171A1
Authority
TN
Tunisia
Prior art keywords
methods
compositions
proteinopathy
mammal
administering
Prior art date
Application number
TNP2015000171A
Other languages
English (en)
French (fr)
Inventor
Seng Cheng
Sergio Pablo Sardi
Lamya Shihabuddin
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=TN2015000171(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 TN2015000171A1 publication Critical patent/TN2015000171A1/fr

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)
  • Veterinary Medicine (AREA)
  • Public Health (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)
  • 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)
  • 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)
TNP2015000171A 2012-11-05 2015-05-04 Compositions and methods for treating proteinopathies TN2015000171A1 (fr)

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
TN2015000171A1 true TN2015000171A1 (fr) 2016-10-03

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
TNP2015000171A TN2015000171A1 (fr) 2012-11-05 2015-05-04 Compositions and methods for treating proteinopathies

Country Status (22)

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

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HUE054957T2 (hu) 2014-09-15 2021-10-28 Orphazyme As Arimoklomol készítése
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
SG11201901265SA (en) * 2015-07-21 2019-03-28 Univ Jefferson Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
MX2018008743A (es) * 2016-01-21 2019-01-30 Protein Dynamic Solutions Llc Método y sistema para el análisis de datos espectrales.
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
RU2021117465A (ru) * 2016-04-29 2021-07-22 Орфазим А/С Аримокломол для лечения ассоциированных с глюкоцереброзидазой нарушений
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
AU2017306558B2 (en) * 2016-08-03 2024-09-19 University Of South Florida Reelin compositions for treatment of neurological disorders
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
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
JP7254815B2 (ja) * 2017-10-03 2023-04-10 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
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
WO2019175883A1 (en) * 2018-03-14 2019-09-19 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
AU2020273182B2 (en) 2019-04-10 2025-11-20 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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
EP4192961A2 (en) 2020-08-06 2023-06-14 Fundacion para la Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
EP4229419A2 (en) * 2020-10-14 2023-08-23 Denali Therapeutics Inc. Methods for treating and monitoring frontotemporal dementia
IL303026A (en) 2020-11-19 2023-07-01 Zevra Denmark As 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
WO2023111335A1 (en) * 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotides capable of increasing glucocerebrosidase expression

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Also Published As

Publication number Publication date
BR112015009746A2 (pt) 2017-08-15
AR093355A1 (es) 2015-06-03
MA38144A1 (fr) 2018-08-31
TW201427695A (zh) 2014-07-16
US20150284472A1 (en) 2015-10-08
AU2013337354A1 (en) 2015-05-21
CN104902923A (zh) 2015-09-09
JP2016503405A (ja) 2016-02-04
HK1214521A1 (zh) 2016-07-29
EA201590880A1 (ru) 2015-09-30
WO2014071282A1 (en) 2014-05-08
ZA201502618B (en) 2016-01-27
PH12015500879A1 (en) 2015-06-29
EP2914281A1 (en) 2015-09-09
MX2015005722A (es) 2016-01-12
CL2015001157A1 (es) 2015-10-16
CR20150216A (es) 2015-05-29
CA2889990A1 (en) 2014-05-08
SG11201502989XA (en) 2015-05-28
KR20150079751A (ko) 2015-07-08
IL238416A0 (en) 2015-06-30

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