Ultragenyx and Andelyn Biosciences are partnering up

Andelyn Biosciences has been chosen by Ultragenyx to produce UX111, the gene therapy for Sanfilippo Syndrome

Andelyn Biosciences, a contract development and manufacturing organization, recently shared that Ultragenyx has chosen them to carry out late-stage process performance qualification (PPQ) manufacturing of UX111. This gene therapy is being developed by Ultragenyx as a potential treatment for Sanfilippo Syndrome (MPS IIIA).

MPS IIIA is caused by a lack of the sulfamidase enzyme. According to a company press release, this condition causes an abnormal buildup of glycosaminoglycans in the brain and body, resulting in cell damage and a decline in neurodevelopment and physical abilities over time.

UX111 is an intravenous infusion that utilizes a self-complementary adeno-associated virus 9 (AAV9) vector to deliver a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells in the central nervous system and peripheral organs. The gene therapy candidate has been granted multiple prestigious designations in both the United States and the European Union, recognizing its potential in regenerative medicine and rare pediatric diseases. These designations include fast track, priority medicine, and orphan drug designations, highlighting the importance and urgency of this innovative treatment.

Andelyn is currently producing a supply of UX111 for patient dosing, following good manufacturing practices. Under this new agreement, the manufacturing of PPQ batches for potential future regulatory filings will commence at the Columbus, Ohio facility.

Source: Andelyn Biosciences