STAT August 24, 2026 • 12:04

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

By Jason Mast

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

The FDA put a hold on a Regenxbio gene therapy trial again after new safety concerns emerged.

The Food and Drug Administration has again paused trials for Regenxbios gene therapy for a rare childhood brain disorder, after small masses were found on the spines of five patients, the company sai… [+699 chars]

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