FDA halts Regenxbio gene therapy trial after spinal masses detected
The FDA has again suspended Regenxbio’s clinical study for a rare childhood brain disorder after imaging revealed small spinal masses in five participants.
Regenxbio announced that five patients with mucopolysaccharidosis type II, also known as Hunter syndrome, were found to have tiny growths on their spines during the trial. The company said none of the children exhibited symptoms related to these masses. This pause follows a prior incident where a boy treated for a similar condition developed a brain tumor, marking the first confirmed tumor linked to AAV gene therapy.
Why it matters
The halt highlights safety uncertainties in emerging gene-therapy approaches for rare diseases.
In this story
FDARegenxbiogene therapyspinal massesHunter syndromeMPS type IIAAVbrain tumorclinical trial
