AVI-4658 is an RNA-based therapeutic employing AVI’s new phosphorodiamidate morpholino oligomer (PMO) based chemistry and exon skipping technologies. It is being developed as a systemic treatment for patients with DMD.
AVI BioPharma has developed AVI-4658, a systemically administered treatment for a substantial subgroup of patients, with Duchenne muscular dystrophy (DMD). The Phase 1b/2 clinical trial in DMD is expected to be initiated in the US this year.
Stephen Shrewsbury, senior vice president and chief medical officer of AVI BioPharma, said: “We are actively working with scientific and medical experts and regulatory authorities to finalise plans for our US based Phase 1b/2 study as we complete the collection and analysis of clinical data from the recent UK trial of AVI-4658.
“The results we have reported to date are promising and suggest an overall favorable safety profile.
“As we continue the clinical evaluation of systemically administered AVI-4658, I remain optimistic about its potential to induce consistent, substantial new dystrophin protein expression in patients with DMD.”