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.
¹Phosphorodiamidate morpholino oligomers
²Candidate received accelerated approval in the U.S., confirmatory studies are ongoing
³Peptide phosphorodiamidate morpholino oligomers
⁴Other exon targets in development: 44, 45, 50, 52, and 53
⁵Roche has the exclusive rights to launch and commercialize SRP-9001 outside the United States
⁶Other LGMD targets in development: SRP-9005 (LGMD2C/R5 γ-sarcoglycan), SRP-9006 (LGMD2L/R12 Anoctamin 5), and Calpain 3 (LGMD2A/R1)