Sarepta’s industry leading pipeline is comprised of over 40 programs in various stages of development across 3 technologies, RNA, gene therapy and gene editing, and multiple therapeutic areas including neuromuscular, CNS and cardiology. Currently, Sarepta has one gene therapy and three RNA-based therapies on the market in the United States.

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Program Details

Information is current as of 4/8/2025, updates are made on a quarterly basis

  • Program Name
  • Discovery/Preclinical
  • Clinical
#661b62
100
SRP-9003
(bidridistrogene xeboparvovec)
#661b62
100
SRP-9004
(patidistrogene bexoparvovec)
#661b62
100
SRP-6004
#661b62
50
Other Targets¹
#172a84
100
SRP-1001 (ARO-DUX4)
#172a84
100
SRP-1003 (ARO-DM1)
#172a84
100
SRP-1004 (ARO-ATXN2)
#172a84
100
SRP-1002 (ARO-MMP7)
#172a84
50
Other Targets²
#8eaa6d
50
CRISPR/CAS9

1 Other gene therapies comprise muscular dystrophy, neuro and cardiac indications including SRP-6006 (LGMD2B/R2 dysferlin), SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), SRP-9010 (LGMD2A/R1 calpain-3-related) and Charcot-Marie-Tooth 

2 Other siRNA indications include Huntington’s disease, SCA1 and SCA3

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