FDA Pauses Regenxbio Gene Therapy Trial After 5 Patients Develop Spinal Masses
Updated
Updated · STAT · Aug 24
FDA Pauses Regenxbio Gene Therapy Trial After 5 Patients Develop Spinal Masses
3 articles · Updated · STAT · Aug 24
Summary
Five trial patients with MPS II, or Hunter syndrome, developed small spinal masses, prompting the FDA to halt Regenxbio’s gene-therapy study again, the company said Monday.
Regenxbio said none of the five patients had symptoms tied to the masses, but the findings add a new safety concern for the treatment.
Seven months ago, the company disclosed that a boy given a closely related MPS I therapy developed a brain tumor.
That case was described as the first tumor conclusively linked to AAV gene therapy, a platform widely used across the field for the past 20 years.
Are viral vectors triggering long-term spinal abnormalities, or is the FDA overreacting to benign growths in rare disease patients?
With a Q3 2026 FDA resubmission looming, will asymptomatic spinal cysts overshadow the cognitive improvements seen in treated Hunter syndrome patients?