Columbus, Ohio, September 18, 2026
Andelyn Biosciences has announced a major milestone in commercial gene therapy manufacturing following the U.S. Food and Drug Administration’s approval of Fayuvi™ (rebisufligene etisparvovec-hopf), an investigational-to-approved gene therapy developed by Ultragenyx Pharmaceutical for Sanfilippo syndrome type A (MPS IIIA). Andelyn is now manufacturing Fayuvi for commercial supply at its Columbus, Ohio facility, making it the first FDA-approved gene therapy manufactured using the company’s AAV Curator® Platform process. The milestone highlights the growing importance of specialized cell and gene therapy manufacturing, commercial-scale viral vector production, and rigorous quality systems in bringing advanced genetic medicines to patients.
Andelyn Biosciences Advances Commercial Gene Therapy Manufacturing
The commercial manufacturing of Fayuvi represents an important step for Andelyn Biosciences as it expands its role from clinical-stage manufacturing into the commercial supply of an FDA-approved gene therapy. The company operates as a cell and gene therapy contract development and manufacturing organization (CDMO), providing services spanning process development, analytical development, plasmid engineering, viral-vector manufacturing, technology transfer, and cGMP clinical and commercial production. According to Andelyn, the Fayuvi milestone demonstrates the scientific, quality, and operational capabilities required to transition a gene therapy program into commercial manufacturing.
Fayuvi is manufactured using Andelyn’s AAV Curator® Platform, a configurable and data-informed approach designed to support adeno-associated virus-based gene therapy programs from development through commercial launch. The platform integrates process knowledge, modular manufacturing strategies, and fit-for-purpose analytics, providing a framework for developing and producing viral vectors while maintaining appropriate quality standards. The company said commercial production of Fayuvi reflects the importance of disciplined process development and cross-functional execution in the gene therapy manufacturing lifecycle.
Fayuvi Approval Highlights Gene Therapy Manufacturing Progress
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The FDA approval of Fayuvi is particularly significant for the treatment of Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). The condition is a rare and fatal lysosomal storage disease that primarily affects the central nervous system and is associated with progressive neurodegeneration beginning in early childhood. Andelyn estimates that MPS IIIA affects approximately 3,000 to 5,000 patients worldwide, with a median life expectancy of about 15 years.
The approval of Fayuvi therefore represents not only a therapeutic milestone but also an important manufacturing achievement for the gene therapy industry. Producing an FDA-approved gene therapy for commercial use requires manufacturing processes, analytical methods, quality systems, facilities, and supply-chain operations capable of consistently supporting product requirements. Andelyn said its commercial manufacturing work for Fayuvi reflects the readiness of its AAV Curator® Platform to support these demands.
AAV Curator Platform Supports Future Gene Therapy Programs
Andelyn Biosciences said its experience extends across more than 20 years, with the company reporting production of more than 500 cGMP batches and support for 85 global clinical trials. Its Columbus operations are designed to support cell and gene therapy programs from early development through clinical manufacturing and commercial production. The company also provides capabilities for both suspension and adherent manufacturing processes, alongside commercial-grade quality systems and vertically integrated supply-chain capabilities. The Fayuvi milestone could further demonstrate the role of specialized CDMOs in gene therapy commercialization, particularly as more AAV-based therapies progress through clinical development and toward regulatory approval.
For biotechnology companies developing genetic medicines, reliable viral-vector manufacturing and scalable cGMP processes remain critical components of successful commercialization. With Fayuvi now approved by the FDA and commercial manufacturing underway at Andelyn’s Columbus facility, Andelyn Biosciences is highlighting the capabilities of its AAV Curator® Platform and its broader role in supporting the growing cell and gene therapy manufacturing ecosystem. The development underscores how advances in manufacturing science, quality systems, and viral-vector production are increasingly connected to the successful delivery of innovative genetic medicines to patients.
Source: Andelyn Biosciences press release
