Andelyn Curator Biomanufacturing Platform Supports Fayuvi Gene Therapy Approval
Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in early childhood. The post Andelyn Curator Biomanufacturing Platform Supports Fayuvi Gene Therapy Approval appeared first on GEN - Genetic Engineering and Biotechnology News .
Andelyn Biosciences has begun manufacturing Fayuvi, an FDA-approved gene therapy for the treatment of Sanfilippo syndrome type A, at its Columbus, OH facility. The FDA recently approved the product developed by Ultragenyx Pharmaceutical for the rare, fatal lysosomal storage disease MPS IIIA, which primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in early childhood.
Fayuvi is the first FDA-approved gene therapy manufactured using Andelyn's AAV Curator Platform process, which utilizes a configurable, data-informed approach to AAV process development and manufacturing. The company's CEO, Wade Macedone, expressed congratulations on this significant achievement and recognition of the MPS IIIA community, investigators, and everyone who contributed to advancing this program.
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