Patent ID: 9133461
Filing Date: 2015-09-15
Classification: A61K,A61P,C12N,C12Y

Abstract:
1. A double-stranded ribonucleic acid (dsRNA) for inhibiting expression of ALAS1, wherein said dsRNA comprises a sense strand and an antisense strand, wherein: (i) the antisense strand is complementary to at least nucleotides 871-889 of SEQ ID NO:1 and comprises SEQ ID NO:1296, (ii) the sense strand comprises at least 15 contiguous nucleotides from SEQ ID NO:1295, (iii) a ligand and linker with a structure as shown below is attached to the 3′ end of the sense strand of the dsRNA and (iv) the dsRNA comprises one or more nucleotides with a modification chosen from a 2′-O-methyl modified nucleotide, a 2′-fluoro modified nucleotide, or both.