The underlying cause of DMD is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function.
Our investigational therapies for DMD are designed to skip an exon in the dystrophin pre-mRNA to enable the synthesis of a shortened form of dystrophin protein.
In addition to our commercial-stage product, candidates for exon 53 skipping (SRP-4053) and exon 45 skipping (SRP-4045) are in clinical development. We also have other drug candidates in discovery and preclinical development that are designed to skip exons 44, 52, 50, 43, 55, 8 and 35, and we are committed to exploring the potential of our technology to address all DMD patients who may be candidates for exon skipping.
EXON SKIPPING: THE BASICS
In DMD, exon skipping is a potential treatment approach to correct for specific genetic mutations and restore production of dystrophin protein.