Exon-Skipping for Duchenne

EXON-SKIPPING FOR DUCHENNE

 
 
 
 
  • PROGRAM
  • DISCOVERY
  • preclinical
  • PHASE I
  • PHASE II
  • PHASE III
  • COMMERCIAL*
 

EXON SKIPPING

  • EXON 51*
  • EXON 53
  • EXON 45
  • EXON 52
  • OTHER EXON TARGETS**
  • PPMO
 
 

*Received accelerated approval in the U.S., confirmatory studies required

**Other exon targets in development: 8, 35, 43, 44, 50, and 55

 

RECENT PRESS RELEASES

     
     
     

    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, functional form of dystrophin protein.

     
     
     
     
     

    Eteplirsen, our lead clinical candidate, is designed to skip exon 51. A candidate for exon 53 skipping (SRP-4053) is in clinical development, and a candidate for exon 45 skipping (SRP-4045) has entered early 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.

    Source: Annemieke Aartsma-Rus, et al. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy. Hum Mutat. 2009 Mar;30 (3):293-9.
    Note: The mutation data highlighted above is derived from the Leiden Duchenne Muscular Dystrophy Mutation Database, which includes a limited dataset.

     
     
     

    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.