STAT+: FDA pauses Regenxbio gene therapy trial again due to safety concerns

Five patients were found to have small masses on their spines Sarah Silbiger/Getty Images 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. The Food and Drug Administration has again paused trials for Regenxbio’s gene therapy for a rare childhood brain disorder, after small masses were found on the spines of five patients, the company said Monday. Regenxbio said the patients, who have mucopolysaccharidosis (MPS) type II or Hunter syndrome, did not have any symptoms related to the masses. The announcement comes seven months after Regenxbio announced that one boy who received a very similar therapy the company developed for MPS type I developed a brain tumor. It was the first tumor to be conclusively linked to AAV gene therapy, the form of gene therapy most commonly used over the past 20 years. STAT+ Exclusive Story Already have an account? Log in 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 View All Plans To read the rest of this story subscribe to STAT+. Subscribe 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.