The candidate, AVI-4658, uses Avi’s exon skipping pre-RNA interference technology (ESPRIT) to potentially benefit Duchenne muscular dystrophy (DMD) patients who have mutations in exon 51 of the dystrophin gene. In animal models, ESPRIT technology has demonstrated the ability to selectively bypass defective exons, restoring near-normal levels of dystrophin production.
Avi plans to initiate a clinical trial with systemically delivered AVI-4658 by mid-2008, to evaluate the safety and potential efficacy of the drug in ambulatory DMD patients.
Michael Forrest, interim CEO of Avi, said: “We expect that Fast Track designation will open the door to expedited clinical development and regulatory review of AVI-4658 in the US.”