FED and the Department of Defense CDMRP program played major roles in promoting the development of exon skipping – the first drug approved for DMD in the US.
FED and CDMRP provided key funding for critical proof-of-principle studies showing that exon skipping could rescue the severe disease of the DMD dogs. Completion of the ground-breaking study required the largest synthesis of morpholino drug ever carried out at that time, at a cost of $1M for enough drug to treat 3 dogs. FED took the plunge, understanding that a proof-of-principle in the large animal model of DMD was critical to move this therapy forward.
FED and CDMRP also carried out critical high dose toxicity studies – to show that morpholino exon skipping drugs were safe at very high levels. FED and CDMRP contributed over $5M to carry out these necessary studies for both exon 51 and exon 45 drugs. Indeed, the FDA approval was based in large part on these studies – where the safety of morpholino drugs was clearly demonstrated.