SRPT (Sarepta Therapeutics Inc.) stock: $17.82, $1.88B market cap, $878.8M cash, $2.02B EV, $2.25B fully diluted market cap. Next expected readout: Nov-26. Lead program: ELEVIDYS (Duchenne Muscular Dystrophy), Phase 4. Data from Fully Diluted's biotech stocks database.
What they do
We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases.
Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Arrowhead Pharmaceuticals: collaboration (annual license fees of $50M; up to $10.3B in development/regulatory/sales milestones; DM1 milestones achieved 2025)
Indications
Myotonic dystrophy type 1(Phase 1)
Catalysts
— H2 2026
Status note Phase 1/2 trial conducted to date by Arrowhead; Sarepta transitioning sponsorship to itself. Early results shared March 2026.
Status note INSIGHTT Phase 1 dose-escalation study (~24 participants); first patient enrolled Q2 2026.
ELEVIDYS (delandistrogene moxeparvovec-rokl)
Approved · ActiveModality: AAV gene therapy
Target DMD
Partnerships Roche: ex-US rights and commercialization
Indications
Duchenne muscular dystrophy (ambulatory)(Stage not disclosed)
Duchenne muscular dystrophy (non-ambulatory)(Stage not disclosed)
Status note Traditional FDA approval (June 2024) for ambulatory patients >=4 years; accelerated approval for non-ambulatory patients (June 2024). Shipments to non-ambulatory patients suspended June 2025; November 2025 boxed warning for acute liver injury/acute liver failure and removal of non-ambulatory population from label. Sirolimus enhanced-immunosuppression study (ENDEAVOR Cohort 8) ongoing to support pathway for resuming non-ambulatory dosing.
SRP-9003
Phase 3 · ActiveModality: AAV gene therapy
Aliases bidridistrogene xeboparvovec
Target SGCB
Indications
LGMD2E (beta-sarcoglycanopathy)(Phase 3)
Status note EMERGENE Phase 3 completed enrollment and dosing December 2024; safety and expression results announced October 2025. FDA clinical hold (July 2025, confirmed December 2025) on investigational LGMD gene therapy trials; BLA acceptance for SRP-9003 requires sirolimus immunosuppressant data first. Other LGMD programs (SRP-9004, SRP-6004, SRP-9005) suspended in July 2025 restructuring.
SRP-9004 / SRP-6004 / SRP-9005
Stage not disclosed · TerminatedModality: AAV gene therapy
Indications
LGMD2D/R4(Stage not disclosed)
LGMD2B/R2(Stage not disclosed)
LGMD2C/R5(Stage not disclosed)
Status note Suspended as part of July 2025 restructuring (following FDA clinical hold after a patient death in the SRP-9004 Phase 1 trial); only SRP-9003 continues.
Duchenne muscular dystrophy (exon 45 amenable)(Stage not disclosed)
Duchenne muscular dystrophy (exon 53 amenable)(Stage not disclosed)
Status note November 2025 confirmatory ESSENCE trial missed its primary endpoint; sNDAs submitted April 2026 and accepted for filing June 2026; FDA discussions on pathway forward ongoing.
Per-asset detail extracted from the 10-Q filed 2026-08-05. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (13 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.