Firm has one candidate in Phase I and another in preclinical studies.
Action Duchenne has earmarked about $1.2 million for a collaboration focused on progressing AVI BioPharma’s exon-skipping drug candidates for the treatment of duchenne muscular dystrophy (DMD).
The company reports that its lead candidate, AVI-4658, is designed to skip exon 51 of the dystrophin gene, restoring the reading frame of the mRNA sequence, and resulting in a truncated but potentially functional form of the dystrophin protein. A dose-finding clinical trial evaluating systemic delivery of AVI-4658 is currently under way in the U.K.
A second, preclinical-stage candidate, AVI-5038, is based on a second generation of AVI BioPharma’s phosphorodiamidate morpholino oligomer antisense chemistry and is deisgned to skip Exon 50.