Research teams at the Imperial College of London, in collaboration with the UK-based MDEX Consortium will conduct the trial of AVI-4658 for AVI in the UK.
The proof-of-principle study 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 improved dystrophin production.
In addition to the UK single-dose IM study, AVI is actively pursuing the expansion of clinical development of AVI-4658 to a multicenter dose-ranging trial using systemic administration of the drug. This trial will be conducted in conjunction with the company’s duchenne muscular dystrophy cross-licensing and development partner, Ercole Biotech.
Michael Forrest, interim CEO of AVI, said: “We are pleased that this clinical trial in DMD is now able to move forward. We are optimistic that our exon skipping pre-RNA interference technology (ESPRIT) approach will be an effective tool in altering the disease mechanism for this and a variety of other diseases, helping the body bypass defective genetic information.”