FDA halts Regenxbio gene therapy trial after spinal masses found in five patients
The Food and Drug Administration paused trials for Regenxbio's gene therapy for mucopolysaccharidosis type II after small masses were found on the spines of five patients, the company said Monday. The patients, who have Hunter syndrome, did not have any symptoms related to the masses, Regenxbio said. The hold comes seven months after the company announced that one boy who received a very similar therapy Regenxbio developed for MPS type I developed a brain tumor. That tumor was the first to be conclusively linked to AAV gene therapy, the form of gene therapy most commonly used over the past 20 years.
This is the second clinical hold for Regenxbio's AAV gene therapy platform in seven months, following the first brain tumor conclusively linked to this widely used form of gene therapy.
Written by the Genomes desk from the primary source cited and linked above and checked against it. Research use only; not medical advice. Corrections: editor@genome.news.