JP2024542790A - プライムエディターの送達のための自己集合性ウイルス様粒子、ならびにその作製および使用方法 - Google Patents

プライムエディターの送達のための自己集合性ウイルス様粒子、ならびにその作製および使用方法 Download PDF

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JP2024542790A
JP2024542790A JP2024533043A JP2024533043A JP2024542790A JP 2024542790 A JP2024542790 A JP 2024542790A JP 2024533043 A JP2024533043 A JP 2024533043A JP 2024533043 A JP2024533043 A JP 2024533043A JP 2024542790 A JP2024542790 A JP 2024542790A
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protein
gag
vlp
nes
fusion
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JP2024542790A5 (https=
Inventor
リウ，デイヴィッド，アール．
ラグラム，アディチャ
バンスコータ，サマギャ
アン，メイルイ
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Broad Institute Inc
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Broad Institute Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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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
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    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
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    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
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    • C07K2319/00—Fusion polypeptide
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
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    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13051—Methods of production or purification of viral material
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    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
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    • C12N2795/18111—Leviviridae
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  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2024533043A 2021-12-03 2022-12-02 プライムエディターの送達のための自己集合性ウイルス様粒子、ならびにその作製および使用方法 Pending JP2024542790A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163285995P 2021-12-03 2021-12-03
US63/285,995 2021-12-03
US202263298626P 2022-01-11 2022-01-11
US63/298,626 2022-01-11
US202263423372P 2022-11-07 2022-11-07
US63/423,372 2022-11-07
PCT/US2022/080836 WO2023102538A1 (en) 2021-12-03 2022-12-02 Self-assembling virus-like particles for delivery of prime editors and methods of making and using same

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Publication Number Publication Date
JP2024542790A true JP2024542790A (ja) 2024-11-15
JP2024542790A5 JP2024542790A5 (https=) 2025-12-10

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JP2024533043A Pending JP2024542790A (ja) 2021-12-03 2022-12-02 プライムエディターの送達のための自己集合性ウイルス様粒子、ならびにその作製および使用方法

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Country Link
US (1) US20250064979A1 (https=)
EP (1) EP4441072A1 (https=)
JP (1) JP2024542790A (https=)
KR (1) KR20240130159A (https=)
AU (1) AU2022403015A1 (https=)
CA (1) CA3239498A1 (https=)
GB (1) GB2630191A (https=)
IL (1) IL313146A (https=)
WO (1) WO2023102538A1 (https=)

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CN118126137A (zh) * 2024-01-19 2024-06-04 微光基因(苏州)有限公司 用于递送核酸和蛋白质的类病毒样蛋白颗粒及其制备方法
WO2025185623A1 (en) * 2024-03-04 2025-09-12 Shanghaitech University Virus-like particles for gene editing
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WO2025248564A1 (en) * 2024-05-30 2025-12-04 Protextent Biosolutions Private Limited A novel virus-like particle platform based on intein-mediated bioconjugation with target antigen and encapsulated with immunomodulatory factors
WO2025262311A1 (en) * 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering
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