Data from RNA editing in humans
Cambridge, US, based Wave Life Sciences Ltd reported positive proof of mechanism data on 16 October from a Phase 1b/2a study of an RNA editing oligonucleotide drug which restored levels of a critical protein in patients with alpha-1 antitrypsin deficiency, a genetic disease affecting the lung and liver. The estimated prevalence of the disease, with the mutation studied in the trial, is 200,000 in the US and Europe.