• Program Name
  • Discovery
  • Preclinical
  • Clinical
  • Commercial*
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87
EXONDYS 51 (eteplirsen)*
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87
VYONDYS 53 (golodirsen)*
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87
AMONDYS 45 (casimersen)*
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40
Exon 52
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20
Exon 43
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20
Exon 44
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20
Exon 50
#661b62
20
Exon 55
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65
SRP-5051
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40
SRP-5053
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40
SRP-5045
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40
SRP-5052
#661b62
40
SRP-5044
#661b62
40
SRP-5050
#661b62
65
SRP-9001 Micro-dystrophin**
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65
GALGT2 (Nationwide Children's)
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65
GNT 0004 (Genethon)
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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)
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40
Calpain 3 (LGMD2A)
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40
Neurotrophin 3 (CMT 1A) (Nationwide Children's)
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65
LYS-SAF302 (MPS IIIA) (Lysogene)
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40
Cardiomyopathy (University of Florida)
#8798e9
40
CNS-1 (Lacerta)
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20
Pompe Disease (Lacerta)
#b25ba3
40
Niemann-Pick Type C (StrideBio)
#999899
20
Rett Syndrome (StrideBio)
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20
Dravet Syndrome (StrideBio)
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20
Angelman Syndrome (StrideBio)
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20
Muscle/CNS Target 1 (StrideBio)
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20
Muscle/CNS Target 2 (StrideBio)
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20
Muscle/CNS Target 3 (StrideBio)
#999899
20
Muscle/CNS Target 4 (StrideBio)
#21bfd1
40
Emery-Dreifuss muscular dystrophy Type 1 (Columbia University)
#6341fa
40
Multiple Sclerosis (University of Florida)
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20
Rett Syndrome (University of Massachusetts)
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20
Duchenne (Institute of Myology)***
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20
CRISPR/CAS9 (Duke University)
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20
Duchenne (Harvard University)

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

** Roche has the exclusive right to launch and commercialize SRP-9001 outside the United States

*** Gene therapy and PPMO

Download PDF of Our Pipeline

For more information about our pipeline, please contact Sarepta’s Patient Affairs team at [email protected].