Andelyn Biosciences begins commercial manufacturing of FDA-Approved Fayuvi Gene Therapy 

Andelyn Biosciences, a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (“CDMO”), announced that 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 US Food and Drug Administration’s approval of Ultragenyx Pharmaceutical Inc.’s Fayuvi for the treatment of 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.

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