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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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (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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US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
EP3292206B8 (fr) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocérébrosidase therapie genique pour la maladie de parkinson
SG11201901265SA (en) * 2015-07-21 2019-03-28 Univ Jefferson Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
EP3405898A1 (fr) * 2016-01-21 2018-11-28 Protein Dynamic Solutions LLC Procédé et système permettant une analyse de données spectrales
WO2017178029A1 (fr) 2016-04-13 2017-10-19 Orphazyme Aps Protéines de choc thermique et homéostasie du cholestérol
CN109069496A (zh) * 2016-04-29 2018-12-21 奥菲泽米有限公司 用于治疗葡糖脑苷脂酶相关疾病的arimoclomol
EP3264092A1 (fr) * 2016-07-01 2018-01-03 Centogene AG Utilisation de gb1 en tant que cible thérapeutique
CN109715194B (zh) * 2016-08-03 2024-06-11 南佛罗里达大学 用于治疗神经障碍的颤蛋白组合物
CN111542549A (zh) * 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
CA3078501A1 (fr) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Therapies geniques pour troubles lysosomaux
EP3692158A4 (fr) 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomiaux
KR20200075865A (ko) * 2017-10-23 2020-06-26 프리베일 테라퓨틱스, 인크. 신경변성 질환에 대한 유전자 요법
KR20240171186A (ko) * 2017-10-26 2024-12-06 다케다 야쿠힌 고교 가부시키가이샤 글루코세레브로시다제 및 이소파고민을 포함하는 제제
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
BR112021019880A2 (pt) * 2019-04-10 2022-02-15 Prevail Therapeutics Inc Terapias gênicas para distúrbios lisossomais
JP7571044B2 (ja) 2019-04-10 2024-10-22 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
MX2021015810A (es) 2019-06-21 2022-08-15 Broad Inst Inc Agentes para revertir las proteinopatias toxicas.
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
JP2023545462A (ja) * 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法
MX2023005954A (es) 2020-11-19 2023-09-04 Zevra Denmark As Procesos para preparar citrato de arimoclomol e intermediarios del mismo.
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
JP2024546925A (ja) * 2021-12-17 2024-12-26 エフ． ホフマン－ラ ロシュ アーゲー グルコセレブロシダーゼ発現を増加させることができるオリゴヌクレオチド

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Also Published As

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

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