COMPOUNDS AND METHODS FOR MODULATION OF SMN2

    公开(公告)号:EP4206213A1

    公开(公告)日:2023-07-05

    申请号:EP22206076.6

    申请日:2017-07-17

    摘要: Disclosed herein are compounds, compositions and compounds for use in modulating splicing of SMN2, said compounds consisting in oligomers comprising a modified oligonucleotide consisting of 14-25 linked nucleosides, wherein the modified oligonucleotide is complementary to an SMN2 pre-mRNA; and wherein at least one nucleoside of the modified oligonucleotide comprises a 2'-O(N-alkyl acetamide) modified sugar moiety. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.