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, a contract manufacturing organization (CMO), has begun producing Fayuvi (rebisufligene etisparvovec-hopf), an FDA-approved gene therapy for commercial distribution. The product, developed by Ultragenyx Pharmaceutical, has been approved for the treatment of Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA).
The FDA recently green-lighted the therapy, which is intended to address the rapid neurodegeneration that typically begins in early childhood in patients with this rare, fatal lysosomal storage disease.
MPS IIIA is estimated to impact between 3,000 and 5,000 patients globally, with a median life expectancy of 15 years. Fayuvi marks the first FDA-approved gene therapy to be manufactured using Andelyn Biosciences' AAV Curator Platform process. Andelyn CEO Wade Macedone expressed his congratulations to Ultragenyx on this notable accomplishment and acknowledged the invaluable contributions of the MPS IIIA community, investigators, and all stakeholders involved in advancing the program.
He emphasized his company's pride in manufacturing an FDA-approved gene therapy for commercial use through their AAV Curator Platform.
Macedone further explained that the AAV Curator Platform employs a flexible, data-driven methodology for AAV process development and manufacturing. This approach integrates process knowledge, modular manufacturing strategies, and tailored analytics to support gene therapy programs from development through commercial launch. The company's manufacturing operation is currently based at its Columbus, Ohio facility, with additional production occurring at Ultragenyx's gene therapy manufacturing site in Bedford, Massachusetts.
Written by urgent.news from GEN Biotechnology's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
