JP2025510739A - リポタンパク質関連疾患を処置するための方法および組成物 - Google Patents

リポタンパク質関連疾患を処置するための方法および組成物 Download PDF

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JP2025510739A
JP2025510739A JP2024556187A JP2024556187A JP2025510739A JP 2025510739 A JP2025510739 A JP 2025510739A JP 2024556187 A JP2024556187 A JP 2024556187A JP 2024556187 A JP2024556187 A JP 2024556187A JP 2025510739 A JP2025510739 A JP 2025510739A
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grna
lpa
sequence
nanoparticles
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JP2025510739A5 (https=
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ドゥソウザ，サンジャイ
ソーワー，ローラ
テレット，ジョナサン
カルマン，ジョン
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CRISPR Therapeutics AG
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CRISPR Therapeutics AG
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Nanotechnology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2024556187A 2022-03-21 2023-03-20 リポタンパク質関連疾患を処置するための方法および組成物 Pending JP2025510739A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202263322078P 2022-03-21 2022-03-21
US63/322,078 2022-03-21
US202263351542P 2022-06-13 2022-06-13
US63/351,542 2022-06-13
US202263385093P 2022-11-28 2022-11-28
US63/385,093 2022-11-28
PCT/IB2023/052720 WO2023180904A1 (en) 2022-03-21 2023-03-20 Methods and compositions for treating lipoprotein-related diseases

Publications (2)

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JP2025510739A true JP2025510739A (ja) 2025-04-15
JP2025510739A5 JP2025510739A5 (https=) 2026-03-25

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JP2024556187A Pending JP2025510739A (ja) 2022-03-21 2023-03-20 リポタンパク質関連疾患を処置するための方法および組成物

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US (4) US20230293646A1 (https=)
EP (1) EP4496891A1 (https=)
JP (1) JP2025510739A (https=)
CN (1) CN119053705A (https=)
AU (1) AU2023240501A1 (https=)
TW (1) TW202403046A (https=)
WO (1) WO2023180904A1 (https=)

Families Citing this family (3)

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CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途
WO2025166325A1 (en) * 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2026008063A1 (en) * 2024-07-05 2026-01-08 Geneditbio Limited Methods and compositions for lpa gene editing and therapy using crispr system

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US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
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CN104072561B (zh) 2007-06-19 2017-12-22 路易斯安那州州立大学及农业机械学院管理委员会 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
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US20250011771A1 (en) 2025-01-09
EP4496891A1 (en) 2025-01-29
US20250144183A1 (en) 2025-05-08
AU2023240501A1 (en) 2024-10-31
US20230293646A1 (en) 2023-09-21
TW202403046A (zh) 2024-01-16
CN119053705A (zh) 2024-11-29
WO2023180904A1 (en) 2023-09-28
US20250228975A1 (en) 2025-07-17

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