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Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

December 19, 2023

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Columbus, OH, December 18, 2023 - Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) 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 and 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. Andelyn 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 EVP, Gene Therapy Research and Development, 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.”

With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

About Andelyn Biosciences, Inc.

Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization, and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn’s deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing curative cell and gene therapies from concept through plasmid development and manufacturing, process development, and cGMP clinical and commercial manufacturing. Andelyn’s versatile capabilities include cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-liter capacity. An advanced digital model, quality system, full regulatory support and supply chain vertical integration help Andelyn accelerate the development and manufacturing of its clients' innovative cell and gene therapies. For more information, visit

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit

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