The FDA put a hold on a Regenxbio gene therapy trial again after new safety concerns emerged.
Five patients were found to have small masses on their spines
Sarah Silbiger/Getty Images
By Jason MastAug. 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.
Investigators detected masses on the spines of five patients who received its Hunter syndrome treatment, leading to the program’s…
The FDA will reconsider approving Regenxbio's experimental gene therapy for a deadly and rare childhood brain disorder it…
Regenxbio plans to submit its Duchenne gene therapy for FDA approval, just a month after it said that regulators wanted to see…
Regenxbio's RGX-121 Hunter syndrome gene therapy faces second clinical hold after spinal lumps found in 5 CAMPSIITE trial…
Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA…
The agency took another step toward clearing a backlog of Makary-era rejections that appeared to contradict earlier agreements…
