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 9/28/2023, updates are made on a quarterly basis

  • Program Name
  • Discovery/Preclinical
  • Clinical
#661b62
100
SRP-5051 (vesleteplirsen)
#661b62
50
Other Exon Targets²
#661b62
100
GNT 0004 - Genethon
#172a84
100
SRP-9003
(bidridistrogene xeboparvovec)
#172a84
100
SRP-9004
(patidistrogene bexoparvovec)
#172a84
100
SRP-6004
#3453a3
50
Other LGMD Targets³
#8eaa6d
50
Other Targets
#661b62
50
CRISPR/CAS9 - Duke University
#661b62
50
CRISPR/CAS9 - Harvard University

¹Peptide phosphorodiamidate morpholino oligomers
²Other exon targets in development: 44, 45, 50, 52, and 53
³Other LGMD targets in development: SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), and SRP-9010 (LGMD2A/R1)

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