In nucleic acid therapeutics, ribose-modified artificial nucleic acids (XNAs) have primarily been used to achieve high affinity for RNA. However, conventional XNAs have backbones structurally similar to those of natural nucleic acids, leaving challenges in resistance to enzymatic degradation and safety. We have developed a novel class of acyclic artificial nucleic acids derived from amino acids, which exhibit high affinity for RNA, excellent resistance to enzymatic degradation, and a favorable safety profile owing to their structurally distinct backbones. We are establishing this technology as a platform for the development of novel nucleic acid therapeutics. Furthermore, leveraging the unique properties of these artificial nucleic acids, we are exploring the development and commercialization of an anti-miR-21 nucleic acid therapeutic for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Hiroyuki Asanuma
Professor, Nagoya univ.