CA3179402A1 - Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease - Google Patents

Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease Download PDF

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Publication number
CA3179402A1
CA3179402A1 CA3179402A CA3179402A CA3179402A1 CA 3179402 A1 CA3179402 A1 CA 3179402A1 CA 3179402 A CA3179402 A CA 3179402A CA 3179402 A CA3179402 A CA 3179402A CA 3179402 A1 CA3179402 A1 CA 3179402A1
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CA
Canada
Prior art keywords
park2
parkin
gene
seq
variant
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.)
Pending
Application number
CA3179402A
Other languages
English (en)
French (fr)
Inventor
Jennifer Johnston
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.)
Nysnobio Gt Neurology LLC
Original Assignee
Nysnobio Gt LLC
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
Application filed by Nysnobio Gt LLC filed Critical Nysnobio Gt LLC
Publication of CA3179402A1 publication Critical patent/CA3179402A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/104—Aminoacyltransferases (2.3.2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/02—Aminoacyltransferases (2.3.2)
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3179402A 2020-05-21 2021-05-21 Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease Pending CA3179402A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063028335P 2020-05-21 2020-05-21
US63/028,335 2020-05-21
PCT/US2021/033758 WO2021237158A1 (en) 2020-05-21 2021-05-21 Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease

Publications (1)

Publication Number Publication Date
CA3179402A1 true CA3179402A1 (en) 2021-11-25

Family

ID=78707645

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3179402A Pending CA3179402A1 (en) 2020-05-21 2021-05-21 Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease

Country Status (6)

Country Link
US (2) US12133897B2 (https=)
EP (1) EP4153760A4 (https=)
JP (1) JP2023526449A (https=)
AU (1) AU2021273979A1 (https=)
CA (1) CA3179402A1 (https=)
WO (1) WO2021237158A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
EP4153760A4 (en) 2020-05-21 2024-08-21 Nysnobio GT, LLC GENE THERAPY ADMINISTRATION OF PARKIN MUTANTS WITH ENHANCED ACTIVITY FOR THE TREATMENT OF PARKINSON'S DISEASE

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1131424B1 (fr) * 1998-11-19 2009-08-19 Aventis Pharma S.A. Mutations du gene de la parkine, compositions, methodes et utilisations
US7998667B1 (en) * 1998-11-19 2011-08-16 Aventis Pharma S.A. Mutations of the parkin gene, compositions, methods and uses
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
WO2009026116A2 (en) * 2007-08-16 2009-02-26 Genizon Biosciences, Inc. Genemap of the human genes associated with longevity
EP2524037B1 (en) * 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US8603994B2 (en) * 2010-11-11 2013-12-10 Valted, Llc Transcriptional repression leading to Parkinson's disease
US9464311B2 (en) 2013-05-02 2016-10-11 E3X Bio, Inc. Method for identifying modulators of ubiquitin ligases
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
MX2020011386A (es) * 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
US12448629B2 (en) * 2018-10-05 2025-10-21 University Of Massachusetts rAAV vectors for the treatment of GM1 and GM2 gangliosidosis
EP4153760A4 (en) 2020-05-21 2024-08-21 Nysnobio GT, LLC GENE THERAPY ADMINISTRATION OF PARKIN MUTANTS WITH ENHANCED ACTIVITY FOR THE TREATMENT OF PARKINSON'S DISEASE

Also Published As

Publication number Publication date
EP4153760A1 (en) 2023-03-29
AU2021273979A1 (en) 2023-01-05
WO2021237158A1 (en) 2021-11-25
US20250049958A1 (en) 2025-02-13
JP2023526449A (ja) 2023-06-21
US12133897B2 (en) 2024-11-05
EP4153760A4 (en) 2024-08-21
US20220111077A1 (en) 2022-04-14

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