Program Name Discovery Preclinical Clinical Commercial* #661B62 87 EXONDYS 51 (eteplirsen)* #661B62 87 VYONDYS 53 (golodirsen)* #661B62 65 Casimersen (SRP-4045) #661B62 40 Exon 52 #661B62 20 Exon 43 #661B62 20 Exon 44 #661B62 20 Exon 50 #661B62 20 Exon 55 #661B62 65 SRP-5051 #661B62 40 SRP-5053 #661B62 40 SRP-5045 #661B62 40 SRP-5052 #661B62 40 SRP-5044 #661B62 40 SRP-5050 #661B62 65 SRP-9001 Micro-dystrophin #661B62 65 GALGT2 (Nationwide Children's) #661B62 40 Micro-dystrophin (Genethon) #3453A3 65 SRP-9003 (LGMD2E β-sarcoglycan) #3453A3 65 SRP-9004 (LGMD2D α-sarcoglycan) #3453A3 40 SRP-9005 (LGMD2C γ-sarcoglycan) #3453A3 65 SRP-6004 (LGMD2B Dysferlin) #3453A3 40 SRP-9006 (LGMD2L Anoctamin 5) #3453A3 40 Calpain 3 (LGMD2A) (Nationwide Children's) #15587B 40 Neurotrophin 3 (CMT 1A) (Nationwide Children's) #8694A4 65 LYS-SAF302 (MPS IIIA) (Lysogene) #8EAA6D 40 Cardiomyopathy (University of Florida) #999899 20 Pompe Disease (Lacerta) #999899 20 CNS-1 (Lacerta) #999899 20 Multiple Sclerosis (University of Florida) #999899 20 Rett Syndrome (University of Massachusetts) #999899 20 Rett Syndrome (StrideBio) #999899 20 Dravet Syndrome (StrideBio) #999899 20 Angelman Syndrome (StrideBio) #999899 20 Niemann-Pick Type C (StrideBio) #999899 20 Muscle/CNS Target 1 (StrideBio) #999899 20 Muscle/CNS Target 2 (StrideBio) #999899 20 Muscle/CNS Target 3 (StrideBio) #999899 20 Muscle/CNS Target 4 (StrideBio) #999899 20 Emery-Dreifuss muscular dystrophy Type 1 (Columbia University) #661B62 20 Duchenne (Institute of Myology)** #661B62 20 CRISPR/CAS9 (Duke University) #661B62 20 Duchenne (Harvard University) * Candidate received accelerated approval in the U.S., confirmatory studies required ** Gene therapy and PPMO Download PDF of Our Pipeline
