JP2023526449A - パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達 - Google Patents

パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達 Download PDF

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JP2023526449A
JP2023526449A JP2022570566A JP2022570566A JP2023526449A JP 2023526449 A JP2023526449 A JP 2023526449A JP 2022570566 A JP2022570566 A JP 2022570566A JP 2022570566 A JP2022570566 A JP 2022570566A JP 2023526449 A JP2023526449 A JP 2023526449A
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park2
parkin
gene
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ジョンストン，ジェニファー
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ニスノバイオ ジーティー，エルエルシー
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    • 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
    • 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
    • A61K48/0075—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
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • 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)
JP2022570566A 2020-05-21 2021-05-21 パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達 Pending JP2023526449A (ja)

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 (2)

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JP2023526449A true JP2023526449A (ja) 2023-06-21
JP2023526449A5 JP2023526449A5 (https=) 2024-04-23

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JP2022570566A Pending JP2023526449A (ja) 2020-05-21 2021-05-21 パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016523517A (ja) * 2013-05-02 2016-08-12 イー３エックス バイオ インコーポレイテッド ユビキチンリガーゼのモジュレーターを同定するための方法
WO2019210325A1 (en) * 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration

Family Cites Families (9)

* 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
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016523517A (ja) * 2013-05-02 2016-08-12 イー３エックス バイオ インコーポレイテッド ユビキチンリガーゼのモジュレーターを同定するための方法
WO2019210325A1 (en) * 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1853, JPN6025011868, 2015, pages 2791 - 2796, ISSN: 0005747760 *
EXPERIMENTAL NEUROLOGY, vol. 207, JPN6025011867, 2007, pages 289 - 301, ISSN: 0005747759 *
HUMAN MOLECULAR GENETICS, vol. 28, no. 17, JPN7025001232, 2019, pages 2811 - 2825, ISSN: 0005747761 *

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Publication number Publication date
EP4153760A1 (en) 2023-03-29
AU2021273979A1 (en) 2023-01-05
WO2021237158A1 (en) 2021-11-25
US20250049958A1 (en) 2025-02-13
US12133897B2 (en) 2024-11-05
EP4153760A4 (en) 2024-08-21
US20220111077A1 (en) 2022-04-14
CA3179402A1 (en) 2021-11-25

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