| abstract |
The present invention relates to a compound having the formula (i) of SEQ ID NO: 1089 or a pharmaceutically acceptable salt thereof. It also refers to a modified oligonucleotide and a conjugated group, wherein the conjugated oligonucleotide is 16 linked nucleosides in length and comprises: a gap segment of 10 linked deoxynucleosides, a 5 'wing segment consisting of three linked nucleosides, and a 3 'wing segment consisting of three linked nucleosides, where the nucleoside comprises a cEt sugar, in which each internucleoside bond is a phosphorothioate bond and each cytosine is 5-methylcytosine, and in which the conjugated group is located in the 5 'end of the modified oligonucleotide and is (ii). It also refers to a pharmaceutical composition. Said compound is an inhibitor of PNPLA3 expression, being useful in the treatment of non-alcoholic fatty liver disease (NAFLD). |