Sarepta’s industry-leading pipeline is comprised of programs in various stages of development. 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-1001 (ARO-DUX4)
#661b62
100
SRP-1003 (ARO-DM1)
#661b62
100
SRP-1004 (ARO-ATXN2)
#661b62
100
SRP-1002 (ARO-MMP7)
#661b62
50
Other Targets¹
#172a84
100
SRP-9003
(bidridistrogene xeboparvovec)

1 Other siRNA indications include Huntington’s disease, spinocerebellar ataxia 1 (SCA1) and spinocerebellar ataxia 3 (SCA3)

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