AU2022402249A1 - Compositions and methods for efficient in vivo delivery - Google Patents

Compositions and methods for efficient in vivo delivery Download PDF

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Publication number
AU2022402249A1
AU2022402249A1 AU2022402249A AU2022402249A AU2022402249A1 AU 2022402249 A1 AU2022402249 A1 AU 2022402249A1 AU 2022402249 A AU2022402249 A AU 2022402249A AU 2022402249 A AU2022402249 A AU 2022402249A AU 2022402249 A1 AU2022402249 A1 AU 2022402249A1
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protein
lipid
ness
containing particle
fusion protein
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AU2022402249A
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Inventor
Meirui AN
Samagya BANSKOTA
Thomas J. CAHILL III
Philip DESOUZA
David R. Liu
Aditya RAGURAM
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Broad Institute Inc
Harvard University
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Broad Institute Inc
Harvard University
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    • C12N15/09—Recombinant DNA-technology
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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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    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/033—Fusion polypeptide containing a localisation/targetting motif containing a motif for targeting to the internal surface of the plasma membrane, e.g. containing a myristoylation motif
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/095—Fusion polypeptide containing a localisation/targetting motif containing a nuclear export signal
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/735—Fusion polypeptide containing domain for protein-protein interaction containing a domain for self-assembly, e.g. a viral coat protein (includes phage display)
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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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    • 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
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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
    • C12N2740/13045—Special targeting system for viral vectors

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AU2022402249A 2021-12-03 2022-12-02 Compositions and methods for efficient in vivo delivery Pending AU2022402249A1 (en)

Applications Claiming Priority (11)

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
US202263298621P 2022-01-11 2022-01-11
US202263298611P 2022-01-11 2022-01-11
US63/298,621 2022-01-11
US63/298,611 2022-01-11
US63/298,626 2022-01-11
US202263423372P 2022-11-07 2022-11-07
US63/423,372 2022-11-07
PCT/US2022/080856 WO2023102550A2 (en) 2021-12-03 2022-12-02 Compositions and methods for efficient in vivo delivery

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AU2022402249A1 true AU2022402249A1 (en) 2024-07-11

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EP (1) EP4441074A2 (enExample)
JP (1) JP2024544013A (enExample)
KR (1) KR20240130158A (enExample)
AU (1) AU2022402249A1 (enExample)
CA (1) CA3239381A1 (enExample)
GB (1) GB2631171A (enExample)
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