• Program
  • Discovery
  • Preclinical
  • Clinical
  • Commercial*
Exondys 51 (Eteplirsen)*
Golodirsen (SRP-4053)
Casimersen (SRP-4045)
Exon 52
Other Exon Targets**
SRP-5051
SRP-5053
SRP-5045
SRP-5052
SRP-5044
SRP-5050
SRP-9001 Micro-Dystrophin
GALGT2 (Nationwide)
Micro-Dystrophin (Genethon)
SRP-9003 (LGMD2E β-sarcoglycan)
SRP-9004 (LGMD2D α-sarcoglycan)
SRP-9005 (LGMD2C γ-sarcoglycan)
MYO-201 (LGMD2B Dysferlin)
SRP-9006 (LGMD2L Anoctamin 5)
CAPN-3 (LGMD2A Calpain 3)
Neurotrophin 3 (CMT 1A)
SRP-9002 (MPS IIIA) (LYSOGENE)***
Pompe Disease
CNS-1 (Lacerta)
CNS-2 (Lacerta)
CRISPR/CAS9 (DUKE UNIVERSITY)