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US 2025/0018028 A1Published application
Self-replicating rna and uses thereof
AssigneeTrustees of Boston University
InventorsJoshua Edward MCGEE, Jack Rainier KIRSCH, Eric BRESSLER, Mark W. GRINSTAFF, Wilson WONG, Lidya Yidnekachew Sertse, Kexin Li
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Abstract
The technology described herein is directed to compositions and methods for modifying and controlling the activity of cells by expression of proteins from self-amplifying RNA (saRNA). Also described herein are compositions and methods for modifying and controlling the activity of cells by expression of proteins from self-amplifying RNA that is substituted with chemically modified nucleotides.
Claims20
A self-amplifying RNA (saRNA) comprising from 5′ to 3′:
- (a) at least one non-structural protein derived from at least one virus;
- (b) a subgenomic promoter (SGP) derived from at least one virus;
- and
- (c) at least one cargo of interest;
- wherein the saRNA comprises at least 25% modified nucleotides, wherein the modified nucleotides are selected from the group consisting of 5-methylcytidine, 5-methyluridine, 5-hydroxymethyluridine, and 5-hydroxymethylcytidine;
- wherein the initiating nucleotide comprises an adenosine or adenosine analog;
- and
- wherein the initiating nucleotide of the saRNA is methylated at the 2′O position of the ribose (Cap1).
5 more elements
A self-amplifying RNA (saRNA) comprising from 5′ to 3′:
- (a) a 5′ cap;
- (b) non-structural protein 1 (nsp1), non-structural protein 2 (nsp2), non-structural protein 3 (nsp3), and/or non-structural protein 4 (nsp4), each derived from at least one virus;
- (c) a subgenomic promoter (SGP) derived from at least one virus;
- (d) a 5′ untranslated region (UTR) derived from at least one virus;
- (e) at least one cargo of interest;
- (f) a 3′ untranslated region (UTR) derived from at least one virus;
- and
- (g) a poly-A tail;
- wherein the cargo comprises a chimeric antigen receptor (CAR) comprising an extracellular domain that specifically binds to an antigen of interest.
6 more elements
Dependencies are inferred from the claim text.