Avidity Biosciences announced that the U.S. FDA has granted Fast Track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping. DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts in males at a very young age. AOC 1044 is being assessed in the Phase 1/2 EXPLORE44 clinical trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. Avidity plans to share results from the healthy volunteer portion of the EXPLORE44 trial in the second half of 2023.
Published first on TheFly
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