Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA) 

Sep 6, 2022 | Clinical Trials, News, Research

In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children.

We are now happy to share that Ultragenyx will be taking over the clinical trials for AAV gene therapy ABO-102 (now UX111).

“Based on promising data from Abeona’s clinical program, regulatory feedback to date, and our experience developing treatments for other MPS diseases, we believe ABO-102 has the potential to be a transformative therapy for patients with MPS IIIA,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Our team’s expertise in MPS and gene therapy clinical development makes this program a seamless integration, and it has the potential to be our first gene therapy to market. The Sanfilippo community has been waiting too long for a first treatment and we believe we can help accelerate this program.”