JP2024523445A - α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 - Google Patents

α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 Download PDF

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JP2024523445A
JP2024523445A JP2023578738A JP2023578738A JP2024523445A JP 2024523445 A JP2024523445 A JP 2024523445A JP 2023578738 A JP2023578738 A JP 2023578738A JP 2023578738 A JP2023578738 A JP 2023578738A JP 2024523445 A JP2024523445 A JP 2024523445A
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rna
seq
syn
aav
sequence
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JP2024523445A5 (https=
JPWO2022268835A5 (https=
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ブロークマンズ， セイダ アッカー
サンチェス， アストリッド バレス
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Uniqure Biopharma BV
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Uniqure Biopharma BV
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023578738A 2021-06-21 2022-06-21 α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用 Pending JP2024523445A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP21180613.8 2021-06-21
EP21180613 2021-06-21
EP22168018 2022-04-12
EP22168018.4 2022-04-12
PCT/EP2022/066922 WO2022268835A1 (en) 2021-06-21 2022-06-21 Gene constructs for silencing alpha-synuclein and uses thereof

Publications (3)

Publication Number Publication Date
JP2024523445A true JP2024523445A (ja) 2024-06-28
JP2024523445A5 JP2024523445A5 (https=) 2025-06-30
JPWO2022268835A5 JPWO2022268835A5 (https=) 2025-06-30

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ID=82358408

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JP2023578738A Pending JP2024523445A (ja) 2021-06-21 2022-06-21 α－シヌクレインをサイレンシングするための遺伝子構築物及びその使用

Country Status (4)

Country Link
US (1) US20250382611A1 (https=)
EP (1) EP4359525A1 (https=)
JP (1) JP2024523445A (https=)
WO (1) WO2022268835A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024337260A1 (en) 2023-09-04 2026-03-12 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles
WO2025078602A1 (en) * 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders
AU2024357870A1 (en) * 2023-10-11 2026-04-23 Uniqure Biopharma B.V. Further novel systems for nucleic acid regulation

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005045034A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
WO2006039253A2 (en) * 2004-09-29 2006-04-13 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
WO2009079399A2 (en) * 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
WO2010129791A1 (en) * 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
HRP20182039T1 (hr) * 2010-04-23 2019-02-08 Cold Spring Harbor Laboratory NOVE STRUKTURNO DIZAJNIRANE MOLEKULE shRNA
CA3077910A1 (en) * 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
PT2744895E (pt) 2011-09-08 2016-02-08 Uniqure Ip Bv Remoção de vírus contaminantes de preparações de aav
AU2015370903B2 (en) 2014-12-24 2021-06-17 Uniqure Ip B.V. RNAi induced huntingtin gene suppression
BR112020006671A2 (pt) * 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
EP3830265A1 (en) * 2018-08-03 2021-06-09 Genzyme Corporation Variant rnai against alpha-synuclein
WO2020104295A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
CA3119721A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
WO2021113270A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing

Also Published As

Publication number Publication date
EP4359525A1 (en) 2024-05-01
WO2022268835A1 (en) 2022-12-29
US20250382611A1 (en) 2025-12-18

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