Ultragenyx's Gene Therapy Milestone: A Turning Point for Sanfilippo Syndrome and the Biotech's Future?
New long-term data for Ultragenyx Pharmaceutical's UX111 gene therapy shows promise in halting cognitive decline in Sanfilippo syndrome type A, coinciding with a key FDA resubmission. While the development marks a significant scientific advance, it unfolds against a backdrop of persistent financial pressures for the company.