FDA halts Regenxbio gene therapy trial after spinal masses found in five patients
The Food and Drug Administration has 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 the rare childhood brain disorder also known as Hunter syndrome, did not have any symptoms related to the masses, Regenxbio said. The announcement comes seven months after one boy who received a very similar therapy Regenxbio developed for MPS type I developed a brain tumor. That 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.
The spinal mass findings add to growing safety concerns around adeno-associated virus gene therapies for central nervous system disorders, following the first confirmed tumor linked to this widely used form of gene therapy.
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].