Program Name Discovery Preclinical Clinical Commercial* #661B6287 EXONDYS 51 (eteplirsen)* #661B6287 VYONDYS 53 (golodirsen)* #661B6287 AMONDYS 45 (casimersen)* #661B6240 Exon 52 #661B6220 Exon 43 #661B6220 Exon 44 #661B6220 Exon 50 #661B6220 Exon 55 #661B6265 SRP-5051 #661B6240 SRP-5053 #661B6240 SRP-5045 #661B6240 SRP-5052 #661B6240 SRP-5044 #661B6240 SRP-5050 #661B6265 SRP-9001 Micro-dystrophin #661B6265 GALGT2 (Nationwide Children's) #661B6265 GNT 0004 (Genethon) #3453A365 SRP-9003 (LGMD2E β-sarcoglycan) #3453A365 SRP-9004 (LGMD2D α-sarcoglycan) #3453A340 SRP-9005 (LGMD2C γ-sarcoglycan) #3453A365 SRP-6004 (LGMD2B Dysferlin) #3453A340 SRP-9006 (LGMD2L Anoctamin 5) #3453A340 Calpain 3 (LGMD2A) (Nationwide Children's) #15587B40 Neurotrophin 3 (CMT 1A) (Nationwide Children's) #8694A465 LYS-SAF302 (MPS IIIA) (Lysogene) #8EAA6D40 Cardiomyopathy (University of Florida) #8798E940 CNS-1 (Lacerta) #99989920 Pompe Disease (Lacerta) #B25BA340 Niemann-Pick Type C (StrideBio) #99989920 Rett Syndrome (StrideBio) #99989920 Dravet Syndrome (StrideBio) #99989920 Angelman Syndrome (StrideBio) #99989920 Muscle/CNS Target 1 (StrideBio) #99989920 Muscle/CNS Target 2 (StrideBio) #99989920 Muscle/CNS Target 3 (StrideBio) #99989920 Muscle/CNS Target 4 (StrideBio) #21BFD140 Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) #6341FA40 Multiple Sclerosis (University of Florida) #99989920 Rett Syndrome (University of Massachusetts) #661B6220 Duchenne (Institute of Myology)** #661B6220 CRISPR/CAS9 (Duke University) #661B6220 Duchenne (Harvard University) * Candidate received accelerated approval in the U.S., confirmatory studies required ** Gene therapy and PPMO Download PDF of Our Pipeline
