Wave’s RNA editor gains proof of concept but not top positioning in AATD

Alpha-1-antitrypsin deficiency is emerging as an early testing ground for emerging genetic modalities, with DNA base editing currently in the lead.

On Wednesday, Wave Life Sciences Inc. (NASDAQ:WVE) reported results from a Phase I/II study of WVE-006, an oligonucleotide-based RNA editor, in patients with alpha-1 antitrypsin deficiency (AATD). The data showed that the RNA editing therapy could restore expression of functional AAT while reducing expression of a damaging mutant version of the protein. ...