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 (enExample
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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
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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
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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
    • 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
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    • 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)
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  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
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  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Virology (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 (2)

Publication Number Publication Date
JP2024523445A true JP2024523445A (ja) 2024-06-28
JP2024523445A5 JP2024523445A5 (enExample) 2025-06-30

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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

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025051805A1 (en) 2023-09-04 2025-03-13 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)
CA2580189C (en) * 2004-09-29 2013-05-21 Children's Memorial Hospital Sirna-mediated gene silencing of alpha synuclein
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
DK2173888T3 (en) 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
US20090176729A1 (en) * 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
JP2011512156A (ja) 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
US9255266B2 (en) * 2009-05-06 2016-02-09 Rutgers, The State University 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
US8993532B2 (en) * 2010-04-23 2015-03-31 Cold Spring Harbor Laboratory Structurally designed shRNAs
CA2817960C (en) * 2010-11-17 2020-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
JP6198278B2 (ja) 2011-09-08 2017-09-20 ユニキュアー アイピー ビー．ブイ． Ａａｖ調製物からの混入ウイルスの除去
HRP20190992T1 (hr) 2014-12-24 2019-09-20 Uniqure Ip B.V. Suprimiranje gena za huntingtin inducirano s rnai
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
KR102931619B1 (ko) * 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
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
AU2020395113A1 (en) * 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing

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

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