Our Pipeline
Explore our upcoming therapies in development
Sarepta's industry-leading pipeline is comprised of programs in various stages of development.
Program Details
Information is current as of April 2026.
Program Name
Discovery / Preclinical
Clinical
siRNA
SRP-1001
Clinical
Facioscapulohumeral muscular dystrophy type 1 (FSHD1)
SRP-1001
Facioscapulohumeral muscular dystrophy type 1 (FSHD1)
SRP-1003
Clinical
Myotonic dystrophy type 1 (DM1)
SRP-1003
Myotonic dystrophy type 1 (DM1)
SRP-1002
Clinical
Idiopathic pulmonary fibrosis (IPF)
SRP-1002
Idiopathic pulmonary fibrosis (IPF)
SRP-1004
Clinical
Spinocerebellar ataxia type 2 (SCA2)
SRP-1004
Spinocerebellar ataxia type 2 (SCA2)
SRP-1005
Clinical
Huntington's disease (HD)
SRP-1005
Huntington's disease (HD)
SRP-1007
Discovery / Preclinical
SCA1*
SRP-1007
SCA1*
SRP-1006
Discovery / Preclinical
SCA3*
SRP-1006
SCA3*
Gene Therapy
SRP-9003
(bidridistrogene xeboparvovec)
Clinical
LGMD2E/R4 β-sarcoglycan
SRP-9003
(bidridistrogene xeboparvovec)
LGMD2E/R4 β-sarcoglycan
* SCA1 = Spinocerebellar ataxia type 1; SCA3 = Spinocerebellar ataxia type 3
