EXONDYS 51
eteplirsen Nucleic Acid Target: dystrophin gene exon 51
Sarepta Therapeutics, Inc.
Most recent annual filing: 2026-03-15
Pipeline (10 active programs)
VYONDYS 53
golodirsen Nucleic Acid Target: dystrophin gene exon 53
AMONDYS 45
casimersen Nucleic Acid Target: dystrophin gene exon 45
ELEVIDYS
delandistrogene moxeparvovec-rokl · SRP-9001 Gene Therapy Target: dystrophin
bidridistrogene xeboparvovec
SRP-9003 Gene Therapy Target: beta-sarcoglycan
SRP-1001
Nucleic Acid Target: DUX4
SRP-1003
Nucleic Acid Target: DMPK
SRP-9004
SRP-6004
Gene Therapy Target: dysferlin
SRP-9005
Gene Therapy Target: gamma-sarcoglycan
Technology platforms
AAVrh74
Adeno-associated virus vector platform using the AAVrh.74 serotype to deliver gene therapy transgenes to target muscle cells; used for Duchenne muscular dystrophy (ELEVIDYS/SRP-9001) and limb-girdle muscular dystrophy programs (SRP-9003). Platform technology designation previously granted by FDA in June 2025, subsequently revoked.
Targets: dystrophin, beta-sarcoglycan
αvβ6 integrin-targeted delivery platform
siRNA delivery platform utilizing αvβ6 integrin targeting for muscle diseases, including facioscapulohumeral muscular dystrophy type 1 (FSHD1) and myotonic dystrophy type 1 (DM1)
Targets: αvβ6 integrin
Biotech Distilled coverage (7)
- 2026-05-06 UPDATE: Sarepta Therapeutics (SRPT) - The Headline Beat That Wasn’t 🔒 Paid
- 2026-04-27 UPDATE: Sarepta Therapeutics (SRPT) - The 10-K Mea Culpa 🔒 Paid
- 2026-03-28 UPDATE: Sarepta Therapeutics (SRPT) - Unsheathing the Sword 🔒 Paid
- 2026-03-21 UPDATE: Sarepta Therapeutics (SRPT) - Fortifying the Shield and Unsheathing the Sword 🔒 Paid
- 2026-03-08 UPDATE: Sarepta Therapeutics (SRPT) - The Other Shoe Drops 🔒 Paid
- 2026-03-01 UPDATE: Sarepta (SRPT) - The Captain Bails Before the Storm? 🔒 Paid
- 2026-02-13 Sarepta Therapeutics (SRPT): Scientific Deep Dive for Lead and Pipeline Products 🔒 Paid
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