US 12,312,586 B2
Treatment of MST1 related diseases and disorders
Omri Gottesman, San Diego, CA (US); Shannon Bruse, San Diego, CA (US); Paul Buske, Madison, WI (US); Brian Cajes, San Diego, CA (US); David Jakubosky, San Diego, CA (US); Sarah Kleinstein, San Diego, CA (US); David Lewis, Madison, WI (US); David Rozema, Cross Plains, WI (US); and John Vekich, San Diego, CA (US)
Assigned to Empirico Inc., San Diego, CA (US)
Filed by Empirico Inc., San Diego, CA (US)
Filed on Aug. 7, 2024, as Appl. No. 18/797,394.
Application 18/797,394 is a continuation of application No. PCT/US2023/083875, filed on Dec. 13, 2023.
Claims priority of provisional application 63/584,461, filed on Sep. 21, 2023.
Claims priority of provisional application 63/582,783, filed on Sep. 14, 2023.
Claims priority of provisional application 63/432,918, filed on Dec. 15, 2022.
Prior Publication US 2024/0392296 A1, Nov. 28, 2024
Int. Cl. C07H 21/04 (2006.01); A61P 11/00 (2006.01); C12N 15/113 (2010.01)
CPC C12N 15/1136 (2013.01) [A61P 11/00 (2018.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/351 (2013.01)] 8 Claims
 
1. A composition
for modulating an expression of MST1 (macrophage-stimulating 1), the composition comprising an oligonucleotide comprising an siRNA comprising a sense strand and an antisense strand,
wherein
each strand is independently 12-30 nucleosides in length;
the sense strand comprises an oligonucleotide sequence of SEQ ID NO: 2999 or 6385; and the antisense strand comprises an oligonucleotide sequence of SEQ ID NO: 6023 or 6415; and
(a) the sense strand comprises modification pattern 30S 5′-snnnnnnNfnNfNfnnnnnnnnnsnsn-3′;
(b) the antisense strand comprises modification pattern 15AS 5′-nsNfsnnnnNfnnNfnNfnNfnNfnNfnsnsn-3′; or
(c) both (a) and (b),
wherein
n is 2′-O-methyl (2′-OMe) A, G, C, and U, respectively;
Nf is 2′-fluoro (2′-F) A, G, C, and U, respectively; and
s is a phosphorothioate linkage.