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. Further, Sarepta is considered a partner of choice and is aligned with some of the best and brightest researchers and companies in the biotechnology industry.
 

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Program Details

Information is current as of 9/29/2022, updates are made on a quarterly basis

  • Program Name
  • Discovery/Preclinical
  • Clinical
  • Commercial
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87
EXONDYS 51 (eteplirsen)²
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87
VYONDYS 53 (golodirsen)²
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87
AMONDYS 45 (casimersen)²
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65
SRP-5051 (vesleteplirsen)
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65
SRP-9001⁵
(delandistrogene moxeparvovec)
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65
GALGT2 - Nationwide Children's
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65
GNT 0004 - Genethon
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65
SRP-9003
(bidridistrogene xeboparvovec)
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65
SRP-9004
(patidistrogene bexoparvovec)
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65
SRP-6004
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20
CRISPR/CAS9 - Duke University
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20
Other Exon Targets⁴
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20
Other LGMD Targets⁶
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20
Other Targets
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20
CRISPR/CAS9 - Harvard University

¹Phosphorodiamidate morpholino oligomers
²Candidate received accelerated approval in the U.S., confirmatory studies are ongoing
³Peptide phosphorodiamidate morpholino oligomers
Other exon targets in development: 44, 45, 50, 52, and 53
Roche has the exclusive rights to launch and commercialize SRP-9001 outside the United States
Other LGMD targets in development: SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), and Calpain 3 (LGMD2A/R1)

Download PDF of Our Pipeline

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