EP4522742A2 — Single-stranded loop oligonucleotides

USPTO Abstract

One aspect of the present invention relates to a single-stranded oligonucleotide having a having a sequence represented by formula (I): (5' - Z 1 - 3')–Q 1 –L–Q 2 –(5' - Z 2 - 3') (I). In formula (I), Z 1 is a first oligonucleotide, comprising 15 – 100 optionally modified nucleotides that is substantially complementary to a target gene; Z 2 is a second oligonucleotide, comprising 15 – 100 optionally modified nucleotides that is substantially complementary to Z 1 ; and Z 1 and Z 2 are capable of forming an intra-strand duplexed region comprising 3 or more consecutive base pairs. L is a linking group. Q 1 and Q 2 each independently represent 0 to 12 optionally modified nucleotides. At least one nucleotide in formula (I) is a modified nucleotide. Other aspects of the invention relate to a pharmaceutical composition and a method for inhibiting the expression of one or more target gene in a subject.