FAYUVI

Columbus, Ohio – September 20, 2026 -- Andelyn Biosciences has begun commercial manufacturing of FAYUVI (rebisufligene etisparvovec-hopf) at its Columbus facility, marking the first FDA-approved gene therapy produced using the company's AAV Curator Platform process. The therapy, developed by Ultragenyx Pharmaceutical Inc., recently received U.S. Food and Drug Administration approval for treating Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA).

FDA clears gene therapy for a rare, fatal childhood disease

MPS IIIA is a rare lysosomal storage disorder that drives rapid neurodegeneration starting in early childhood, primarily affecting the central nervous system. The disease impacts an estimated 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years.