FDA rare pediatric disease designation for Avidity’s AOC 1044

US RNA-focussed biotech Avidity Biosciences (Nasdaq: RNA) says that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, the company’s investigational therapy in development for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44).

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 at a very young age. Currently, there are no approved therapies targeting exon 44.

Avidity explained that AOC 1044 is based on the company’s AOC platform that combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target tissues/cells previously unreachable with existing RNA therapies. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in patients.