RNA Editing

RNA editing offers reversible alternatives to DNA editing with growing clinical-trial activity. The session covers ADAR-recruiting oligonucleotides (Wave Life Sciences, Korro Bio, ProQR Therapeutics pipelines), the comparison with CRISPR DNA editing, first-in-human RNA editing programmes, the regulatory pathway for the first RNA-editing therapies, RNA editing in alpha-1 antitrypsin deficiency and other liver-targeted indications, and the technology evolution from ADAR1 to engineered editors. Discussion addresses delivery challenges for RNA-editing therapeutics, the immunogenicity considerations, the IP landscape, the comparison of programmable vs constitutive RNA editing, and the role of CRISPR-Cas13 RNA-targeting platforms.