The trial will include up to nine boys with Duchenne muscular dystrophy (DMD), each of whom will receive a single intramuscular (IM) administration of the drug. Two to three weeks following the injection, the muscle will be biopsied and examined for molecular evidence of corrected dystrophin production. AVI-4658 is based on the company’s proprietary ESPRIT (exon skipping pre-RNA interference technology) drug platform.
Michael Forrest, interim CEO of AVI, said: “We are pleased that this clinical trial in DMD is now moving forward. We are simultaneously moving toward initiating clinical trials evaluating longer-term systemic administration of AVI-4658.”