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news.AVI BioPharma has dosed the first patient in a proof-of-principle clinical trial using AVI-4658, AVI's lead drug candidate for Duchenne muscular dystrophy.
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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.”