Our Pipeline

In addition to our FDA-approved treatment for Duchenne muscular dystrophy, we are developing a variety of drug candidates based on our proprietary RNA-based technology and unique phosphorodiamidate morpholino oligomer – or PMO – chemistry.

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

EXON SKIPPING

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

GENE THERAPY

  • MICRO-DYSTROPHIN
  • GALGT2
 

UTROPHIN MODULATION

  • EZUTROMID
 
 
 

Internal


External Collaborations

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

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