Regenxbio reported Monday the first clinical evidence showing improved muscle function in boys with Duchenne muscular dystrophy following treatment with its experimental gene therapy.
The positive results from muscle-performance tests come from a small number of patients, but after consulting with the Food and Drug Administration, Regenxbio has expanded its current clinical trial so that it can support the submission of an accelerated approval application in 2026.
If successful, the Regenxbio gene therapy, called RGX-202, could become the second genetic medicine for Duchenne to reach the market, following the accelerated approval of Sarepta Therapeutics’ Elevidys in June 2023. It might also become the first Duchenne gene therapy accessible to children starting on their first birthday. Elevidys can’t be given to children under 4.
This article is exclusive to STAT+ subscribers
Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.
Already have an account? Log in
