FDA Pauses Regenxbio Gene Therapy Trial Over Spinal Masses
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- Regenxbio disclosed that the FDA paused its gene therapy trial for mucopolysaccharidosis type II (Hunter syndrome) after small masses appeared on the spines of five patients
- Regenxbio said the five patients with spinal masses showed no symptoms related to the growths
- Regenxbio announced the pause roughly seven months after a boy receiving the company's similar MPS type I therapy developed a brain tumor
- The brain tumor case marked the first tumor conclusively linked to AAV gene therapy, the delivery method most commonly used in the field over the past 20 years
Why it matters: For Regenxbio and the broader AAV gene therapy field, a second safety pause within seven months — and the first-ever confirmed tumor tied to AAV delivery — puts added regulatory scrutiny on a modality that has powered the majority of gene therapy development for two decades. Five spinal findings across MPS II patients give the FDA a concrete cluster to evaluate, even if the company reports no symptoms.
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