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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)
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)
  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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 (fr) 2012-11-05 2013-11-04 Compositions et procédés pour le traitement de protéinopathies

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

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RU2013125923A (ru) 2010-11-30 2015-01-10 Орфазиме Апс СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70
CA2961097C (fr) 2014-09-15 2023-09-26 Orphazyme Aps Formulation pharmaceutique a liberation prolongee comprenant de arimoclomol
CA2985235A1 (fr) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Therapie genique par glucocerebrosidase pour la maladie de parkinson
CA3030261A1 (fr) * 2015-07-21 2017-01-26 Thomas Jefferson University Therapies geniques pour les troubles neurodegeneratifs ciblant les voies de biosynthese des gangliosides
EP3442530A1 (fr) 2016-04-13 2019-02-20 Orphazyme A/S Protéines de choc thermique et homéostasie du cholestérol
DK3448382T3 (da) * 2016-04-29 2020-12-07 Orphazyme As Arimoclomol til behandling af glucocerebrosidase-associerede lidelser
EP3264092A1 (fr) * 2016-07-01 2018-01-03 Centogene AG Utilisation de gb1 en tant que cible thérapeutique
JP7253800B2 (ja) * 2016-08-03 2023-04-07 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
KR20200078513A (ko) 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
EP3692075A4 (fr) * 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux
WO2019070894A1 (fr) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomiaux
AU2018354195A1 (en) 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CA3078463A1 (fr) * 2017-10-26 2019-05-02 Shire Human Genetic Therapies, Inc. Formulations comprenant de la glucocerebrosidase et de l'isofagomine
EP3765855A1 (fr) * 2018-03-14 2021-01-20 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd Détection de phospho-sérine 129 alpha-synucléine dans des cellules sanguines en tant que biomarqueur pour synucléinopathies
WO2020210713A1 (fr) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux
EP3953377A1 (fr) 2019-04-10 2022-02-16 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux
MX2021015810A (es) 2019-06-21 2022-08-15 Broad Inst Inc Agentes para revertir las proteinopatias toxicas.
MX2023000968A (es) 2020-08-06 2023-03-01 Fundacion Para La Investig Medica Aplicada Particulas virales para usarse en el tratamiento contra tauopatias tales como enfermedad de alzheimer mediante terapia genica.
EP4247792A1 (fr) 2020-11-19 2023-09-27 Zevra Denmark A/S Procédés de préparation de citrate d'arimoclomol et intermédiaires associés
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
CN118382697A (zh) * 2021-12-17 2024-07-23 豪夫迈·罗氏有限公司 能够增加葡糖脑苷脂酶表达的寡核苷酸

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Also Published As

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

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