Sarepta Therapeutics

SRPT Rare disease Lead: Approved 10 Biotech Distilled articles ↓

Most recent annual filing: 2026-03-15

Catalyst timeline

ELEVIDYS ENDEAVOR readout2H 2026ELEVIDYS ENDEAVOR readout, 2H 2026, major
Catalent clean room agreement corporate4Q 2026Catalent clean room agreement corporate, 4Q 2026, routine
ELEVIDYS readout1Q 2027ELEVIDYS readout, 1Q 2027, major

Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.

Pipeline (10 active programs)

eteplirsen

EXONDYS 51

Nucleic Acid Target: dystrophin gene exon 51, dystrophin exon 51

Duchenne Muscular Dystrophy Approved intravenous NCT01396239

golodirsen

VYONDYS 53

Nucleic Acid Target: dystrophin gene exon 53, dystrophin exon 53

Duchenne Muscular Dystrophy Approved intravenous NCT04708314

casimersen

AMONDYS 45

Nucleic Acid Target: dystrophin gene exon 45, dystrophin exon 45

Duchenne Muscular Dystrophy Approved intravenous

ELEVIDYS

delandistrogene moxeparvovec-rokl · SRP-9001

Gene Therapy Target: dystrophin, dystrophin gene

Duchenne Muscular Dystrophy Approved Roche NCT03375164

bidridistrogene xeboparvovec

SRP-9003

Gene Therapy Target: beta-sarcoglycan

Limb-Girdle Muscular Dystrophy Phase 3 NCT03652259

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

Discontinued (3)

SRP-9004 — Limb-Girdle Muscular Dystrophy · suspended as part of Restructuring; FDA clinical hold placed following patient death
SRP-6004 — Limb-Girdle Muscular Dystrophy · suspended as part of Restructuring; FDA clinical hold placed
SRP-9005 — Limb-Girdle Muscular Dystrophy · suspended as part of Restructuring; FDA clinical hold placed

Biotech Distilled coverage (10)

2026-08-06 UPDATE: Sarepta Therapeutics (SRPT) - The Clock Slips a Quarter 🔒 Paid
2026-07-28 UPDATE: Sarepta Therapeutics (SRPT) - The Chair Gets Filled 🔒 Paid
2026-06-30 UPDATE: Sarepta Therapeutics (SRPT) - The FDA Took the Meeting
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

Articles open on Substack. Some deep dives are subscriber-only.