COLUMBUS, Ohio, Sept. 18, 2026 /PRNewswire/ — Andelyn Biosciences (“Andelyn”), a leading, patient-focused cell and gene therapy Contract Development and Manufacturing Organization (“CDMO”), today announced it is now manufacturing FAYUVI™ (rebisufligene etisparvovec-hopf), an FDA-approved gene therapy, for commercial supply at its Columbus, Ohio facility. The announcement follows the U.S. Food and Drug Administration’s approval of Ultragenyx Pharmaceutical Inc.’s FAYUVI™ for treating Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). 

The approval is a significant milestone for the MPS IIIA community and for families affected by the disease, and it reflects the sustained commitment of the Ultragenyx team, clinical investigators, patient advocates, and others who helped advance the program.

Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system (CNS) and is marked by rapid neurodegeneration beginning in early childhood. MPS IIIA is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years.

FAYUVI™ is the first FDA-approved gene therapy manufactured using the Andelyn AAV Curator® Platform process. Its commercial manufacturing at Andelyn’s Columbus facility reflects the disciplined process development, scientific and quality systems rigor, and cross-functional execution required to bring a gene therapy program to market.

“We congratulate Ultragenyx on this significant achievement and recognize the MPS IIIA community, investigators, and everyone who contributed to advancing this program,” said Wade Macedone, Chief Executive Officer of Andelyn Biosciences. “We are proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator® Platform process. This milestone reflects the purpose behind Andelyn’s founding and underscores the importance of building commercial readiness through rigorous science, accountable execution, and quality at every stage. We look forward to supporting our other late-stage clients and the industry with our FDA-stamped commercial capabilities.”

Andelyn’s AAV Curator® Platform uses a configurable, data-informed approach to AAV process development and manufacturing. It integrates process knowledge, modular manufacturing strategies, and fit-for-purpose analytics to support gene therapy programs from development through commercial launch.

About Andelyn Biosciences

Andelyn Biosciences is a full-service FDA-inspected cell and gene therapy commercial CDMO focused on developing, characterizing, and producing viral vectors for gene therapy. With more than 20 years of experience, Andelyn’s deep scientific expertise has resulted in the production of clinical and commercial material for more than 500 cGMP batches and 85 global clinical trials, including an FDA-approved gene therapy. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high-quality products by developing and manufacturing processes on its configurable, data-driven Curator® Cell and Gene Therapy Platforms, or by tech transfer within an established client program. Capabilities include cGMP manufacturing for suspension and adherent processes. A rigorous, commercial-grade quality system, phase-appropriate manufacturing, and supply chain vertical integration further help clients bring critical therapies to market.

For more information, please visit andelynbio.com and follow Andelyn Biosciences on LinkedIn.

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SOURCE Andelyn Biosciences