Sarepta Therapeutics’ share price has fallen again following the death of a third patient dosed with its gene therapy treatment.
A 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 died after receiving Sarepta’s gene therapy candidate SRP-9004 in a Phase I trial, according to Bloomberg.
Sarepta Therapeutics was approached for comment by Clinical Trials Arena but did not respond before publication.
This comes after two deaths were announced following treatment with Sarepta’s Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec). Both patient deaths, the first announced in March 2025 and the second in June 2025, were due to acute liver failure (ALF).
Elevidys and SRP-9004 both use the same recombinant adeno-associated viral vector (AAV) in AAVrh74.
Reports state that Sarepta has informed regulators and clinical investigators of the latest death after dosing with SRP-9004.
Earlier this week, Sarepta said it was cutting 500 jobs as part of its restructuring and pipeline prioritisation plan, as well as updating the label for Elevidys. The company is engaging with the US Food and Drug Administration (FDA) regarding the label update for the gene therapy, which includes adding a black box warning for acute liver injury (ALI) and ALF.
Nasdaq-listed Sarepta saw its share price jump 19.53%, rising from $18.38 at the close of 16 July to $21.97 on July 17, following news of the restructuring and new Elevidys label. However, shares plunged 22.62%, to open at $17.00 on 18 July after the company announced the death of the patient in the clinical trial.
In May 2025, the FDA announced that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA’s decision to approve the therapy.
CGT sector at a “crossroads”
GlobalData healthcare analyst Momna Ali said the recent setbacks for Sarepta put the cell and gene therapy (CGT) sector at a “crossroads”.
Ali said: “Following Sarepta’s announcement of a third patient death, a new label for Elevidys and them laying off 500 employees, or 36% of its workforce, shelving parts of its pipeline to save $420m, and shifting focus from gene therapy to siRNA programmes, this is going to put the CGT sector at a pivotal crossroad.
"While the scientific potential of CGT therapies remains extraordinary, this moment serves as a reminder of the complexity, cost, and responsibility involved. There is a lot of buzz around therapies ‘beyond the pill'; however, for the landscape to keep evolving at the pace it has been in the last 3-5 years, there has to be greater transparency, patient safety, and sustainable innovation - otherwise, it’ll be met with more setbacks.”
Pfizer pulled an investigational DMD therapy last year after the death of a patient in a Phase II trial and a Phase III trial failed to meet its primary endpoint.
In May 2025, a patient in Rocket Pharmaceuticals’ pivotal Phase II trial of gene therapy for Danon disease died after suffering a serious adverse event (AE).
