Ultragenyx Picks Andelyn Biosciences to Manufacture Novel Gene Therapy for Sanfilippo Syndrome

Published on: 

Andelyn Biosciences has been selected by Ultragenyx to manufacture UX111, that company’s gene therapy for treating Sanfilippo Syndrome.

On Dec. 18, 2023, Andelyn Biosciences, a contract development and manufacturing organization, announced that Ultragenyx has selected it to perform late-stage process performance qualification (PPQ) manufacturing of UX111, Ultragenyx’s investigational gene therapy under development for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

MPS IIIA is related to a deficiency of the sulfamidase enzyme. This deficiency is responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body, which leads to progressive cell damage and neurodevelopmental and physical decline, according to a company press release.

UX111 is a one-time intravenous infusion that uses a self-complementary adeno-associated virus 9 (AAV9) vector to deliver a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to central nervous system cells and peripheral organs. The gene therapy candidate has received regenerative medicine advanced therapy, fast track, rare pediatric disease, and orphan drug designations in the United States, and priority medicine and orphan medicinal product designations in the European Union.


Currently, Andelyn has been manufacturing good manufacturing practice supply of UX111 for patient dosing. With this new agreement, it will begin manufacturing PPQ batches that may be required for future regulatory filings at its Columbus, Ohio, facility.

Source: Andelyn Biosciences