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

December 27, 2023

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.

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