FDA pauses Regenxbio gene therapy trial again after spinal masses found in five patients

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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.

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