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.
Exondys 51 (Eteplirsen)*
Other Exon Targets**
CRISPR/CAS9 (DUKE UNIVERSITY)
*Candidate received accelerated approval in the U.S., confirmatory studies required
**Other exon targets in development: 8, 35, 43, 44, 50, and 55