Regenxbio plans to submit its Duchenne gene therapy for FDA approval, just a month after it said that regulators wanted to see another trial.
The agency has recently signaled it’s more open to considering rare disease treatments after a staff shakeup
Alex Hogan/STAT
By Jason MastJune 24, 2026
General Assignment Reporter
Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.
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Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA…
Regenxbio said its experimental gene therapy for Duchenne muscular dystrophy succeeded in a trial, paving the way for a…
Regenxbio said Thursday that its Duchenne muscular dystrophy gene therapy met the bar in a pivotal study, and it’s aiming for FDA…
The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it…
Regenxbio plans to resubmit its Hunter syndrome gene therapy RGX-121 after the FDA reversed course, saying existing data are…
FDA reverses its rejection of Regenxbio Navsunli, allowing the company to refile its gene therapy application.
