CA2889990A1 - Compositions and methods for treating proteinopathies - Google Patents

Compositions and methods for treating proteinopathies Download PDF

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Publication number
CA2889990A1
CA2889990A1 CA2889990A CA2889990A CA2889990A1 CA 2889990 A1 CA2889990 A1 CA 2889990A1 CA 2889990 A CA2889990 A CA 2889990A CA 2889990 A CA2889990 A CA 2889990A CA 2889990 A1 CA2889990 A1 CA 2889990A1
Authority
CA
Canada
Prior art keywords
aav
disease
gba1
agent
synuclein
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA2889990A
Other languages
English (en)
French (fr)
Inventor
Sergio Pablo SARDI
Lamya Shihabuddin
Seng Cheng
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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=CA2889990(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 CA2889990A1 publication Critical patent/CA2889990A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IG], 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 [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], 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)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2889990A 2012-11-05 2013-11-04 Compositions and methods for treating proteinopathies Abandoned CA2889990A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261722434P 2012-11-05 2012-11-05
US61/722,434 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
CA2889990A1 true CA2889990A1 (en) 2014-05-08

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2889990A Abandoned CA2889990A1 (en) 2012-11-05 2013-11-04 Compositions and methods for treating proteinopathies

Country Status (22)

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

Cited By (1)

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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

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AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
SG10202100652VA (en) * 2015-07-21 2021-03-30 Univ Jefferson Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
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US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
DK3782624T3 (da) * 2016-04-29 2025-12-15 Zevra Denmark As Arimoclomol til behandling af glucocerebrosidase-associerede lidelser
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EP3506927A4 (en) 2016-08-03 2020-05-06 University of South Florida REELIN COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASES
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
EP3692075A4 (en) * 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN111278439A (zh) * 2017-10-26 2020-06-12 夏尔人类遗传性治疗公司 包含葡萄糖脑苷脂酶和异烟肼的调配物
CA3136117A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. Agents for reversing 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
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
WO2022082178A2 (en) * 2020-10-14 2022-04-21 Denali Therapeutics Inc. Methods for treating and monitoring frontotemporal dementia
KR20230128462A (ko) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 아리모클로몰 시트레이트 및 이의 중간체를 제조하기 위한 공정
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
EP4448763A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotide gba agonists
WO2023111335A1 (en) * 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Oligonucleotides capable of increasing glucocerebrosidase expression

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
EP2914281A1 (en) 2015-09-09
SG11201502989XA (en) 2015-05-28
CR20150216A (es) 2015-05-29
BR112015009746A2 (pt) 2017-08-15
CN104902923A (zh) 2015-09-09
CL2015001157A1 (es) 2015-10-16
JP2016503405A (ja) 2016-02-04
TW201427695A (zh) 2014-07-16
WO2014071282A1 (en) 2014-05-08
ZA201502618B (en) 2016-01-27
AR093355A1 (es) 2015-06-03
MX2015005722A (es) 2016-01-12
KR20150079751A (ko) 2015-07-08
IL238416A0 (en) 2015-06-30
EA201590880A1 (ru) 2015-09-30
US20150284472A1 (en) 2015-10-08
MA38144A1 (fr) 2018-08-31
PH12015500879A1 (en) 2015-06-29
AU2013337354A1 (en) 2015-05-21
HK1214521A1 (zh) 2016-07-29
TN2015000171A1 (fr) 2016-10-03

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FZDE Discontinued

Effective date: 20181106