US 12,115,217 B2
Self-replicating RNA and uses thereof
Joshua Edward McGee, Watertown, MA (US); Jack Rainier Kirsch, Cambridge, MA (US); Eric Bressler, Boston, MA (US); Mark W. Grinstaff, Brookline, MA (US); Wilson Wong, Brookline, MA (US); Lidya Yidnekachew Sertse, Boston, MA (US); and Kexin Li, Boston, MA (US)
Assigned to Trustees of Boston University
Filed by Trustees of Boston University, Boston, MA (US)
Filed on Nov. 17, 2023, as Appl. No. 18/512,730.
Claims priority of provisional application 63/538,540, filed on Sep. 15, 2023.
Claims priority of provisional application 63/460,506, filed on Apr. 19, 2023.
Claims priority of provisional application 63/426,597, filed on Nov. 18, 2022.
Prior Publication US 2024/0197860 A1, Jun. 20, 2024
Int. Cl. A61K 39/00 (2006.01); A61K 39/02 (2006.01); A61K 39/12 (2006.01); A61K 39/155 (2006.01); A61K 39/215 (2006.01); A61P 31/04 (2006.01); A61P 37/04 (2006.01); C07K 14/005 (2006.01); C07K 14/575 (2006.01); C07K 16/28 (2006.01); C07K 16/32 (2006.01); C12N 15/86 (2006.01)
CPC A61K 39/215 (2013.01) [A61K 39/4631 (2023.05); A61P 37/04 (2018.01); C07K 14/005 (2013.01); C07K 14/575 (2013.01); C07K 16/2803 (2013.01); C07K 16/32 (2013.01); C12N 15/86 (2013.01); A61K 2039/53 (2013.01); A61K 2239/23 (2023.05); C12N 2770/20022 (2013.01); C12N 2770/36143 (2013.01); C12N 2830/002 (2013.01); C12N 2830/50 (2013.01); C12N 2840/203 (2013.01)] 30 Claims
 
1. A self-amplifying RNA (saRNA) comprising at least 25% modified nucleotides, wherein the modified nucleotides comprise a pyrimidine nucleoside phosphate with a moiety on carbon 5 of the pyrimidine, wherein the moiety is selected from the group consisting of methyl, ethyl, propyl, trifluoromethyl, hydroxymethyl, hydroxyethyl, and hydroxypropyl functional groups, and at least one cargo of interest.