Treatment of MST1 related diseases and disorders
AssigneeEmpirico Inc.
InventorsOmri Gottesman, Shannon Bruse, Paul Buske, Brian Cajes, David Jakubosky, Sarah Kleinstein, David Lewis, David Rozema, John Vekich
Life of this patent
Estimating the term…
Abstract
Disclosed herein are compositions comprising an oligonucleotide that targets MST1. The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating conditions associated with MST1 variants that include providing an oligonucleotide that targets MST1 to a subject.
Claims8
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.
A composition
- for modulating an expression of MST1 (macrophage-stimulating 1), the composition comprising a modified oligonucleotide comprising an siRNA comprising a sense strand and an antisense strand, each strand is independently 12-30 nucleosides in length, at least one of the sense strand and the antisense strand comprises a nucleoside sequence comprising 12-30 contiguous nucleosides of SEQ ID NO:
- 6185;
- 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.
A double stranded ribonucleic acid (dsRNA) for inhibiting expression of MST1 (macrophage-stimulating 1) in a cell, or a pharmaceutically acceptable salt thereof, comprising:
- a sense strand and an antisense strand forming a double stranded region, wherein the sense strand comprises the nucleotide sequence:
- 5′-[ETL17]sacuucuUfgUfCfagacauaaasusu-3′ (SEQ ID NO:
- 6538), and wherein the antisense strand comprises the nucleotide sequence:
- 5′-usUfsuaugUfcuGfaCfaAfgAfaGfususu-3′ (SEQ ID NO:
- 6570), wherein
- a, g, c, and u are 2′-O-methyl (2′-OMe) A, G, C, and U, respectively;
- Af, Gf, Cf, and Uf are 2′-fluoro (2′-F) A, G, C, and U, respectively;
- s is a phosphorothioate linkage, and
- [ETL17] is
- wherein J is the attachment point to the 5′ phosphorothioate linkage of the sense strand.
Dependencies are inferred from the claim text.