GB2630191A - Self-assembling virus-like particles for delivery of prime editors and methods of making and using same - Google Patents

Self-assembling virus-like particles for delivery of prime editors and methods of making and using same Download PDF

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Publication number
GB2630191A
GB2630191A GB2409668.7A GB202409668A GB2630191A GB 2630191 A GB2630191 A GB 2630191A GB 202409668 A GB202409668 A GB 202409668A GB 2630191 A GB2630191 A GB 2630191A
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United Kingdom
Prior art keywords
gag
nes
protein
vlp
polynucleotides
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GB2409668.7A
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GB202409668D0 (en
Inventor
R Liu David
Raguram Aditya
Banskota Samagya
An Meirui
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Broad Institute Inc
Harvard University
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Broad Institute Inc
Harvard University
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Publication of GB202409668D0 publication Critical patent/GB202409668D0/en
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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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
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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
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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
    • C12N15/86—Viral vectors
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12Y—ENZYMES
    • 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
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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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
    • C12N2740/13022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13023—Virus like particles [VLP]
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    • 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
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • 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
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    • C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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GB2409668.7A 2021-12-03 2022-12-02 Self-assembling virus-like particles for delivery of prime editors and methods of making and using same Pending GB2630191A (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163285995P 2021-12-03 2021-12-03
US202263298626P 2022-01-11 2022-01-11
US202263423372P 2022-11-07 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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GB202409668D0 GB202409668D0 (en) 2024-08-14
GB2630191A true GB2630191A (en) 2024-11-20

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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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WO2025051799A1 (en) 2023-09-05 2025-03-13 Aarhus Universitet Dna-encoded fusion proteins for incorporation of prime editing protein/rna complexes in virus-like particles
WO2025096936A2 (en) 2023-11-03 2025-05-08 The Broad Institute, Inc. Use of prime editing in correcting mutations in cdkl5
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
WO2025217616A1 (en) 2024-04-12 2025-10-16 The Broad Institute, Inc. Prime editing and base editing of the atp1a3 gene for the treatment of alternating hemiplegia of childhood
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
WO2025262314A1 (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
WO2026024856A2 (en) 2024-07-23 2026-01-29 The Broad Institute, Inc. Compositions and methods for production of double-stranded dna in cells for programmable gene integration
CN121628933A (zh) * 2024-08-30 2026-03-10 中国科学院遗传与发育生物学研究所 反向引导编辑系统

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