Andelyn Biosciences, Inc., a cell and gene therapy CDMO, has been selected by Ultragenyx to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company’s UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

MPS IIIA is derived from a sulfamidase enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body leading to progressive cell damage and neurodevelopmental and physical decline. Ultragenyx’s UX111 investigational gene therapy is a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. It has received regenerative medicine advanced therapy, fast track, rare pediatric disease, and orphan drug designations in the U.S., and PRIME and orphan medicinal product designations in the EU.

In a statement, Andelyn said that it has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.

Matt Niloff, chief commercial officer of Andelyn, said, “Working collaboratively with Ultragenyx and leveraging our 20 years of AAV experience, we have successfully transferred the UX111 GMP process to our commercial manufacturing facility in Columbus, Ohio.  Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner.”

Dennis Huang, chief technical operations officer and executive vice president, gene therapy research and development, Ultragenyx, said, “Working with Andelyn was a great choice given their history with the product, their extensive multi-product experience with the manufacturing technology used for the product, and our desire to quickly bring UX111 to MPS IIIA patients.”