Gene Therapy Clinical Trial for Sanfilippo Syndrome Type B (MPS IIIB) is Underway

Abeona Therapeutics, Inc. is enrolling individuals aged 6 months and older who have MPS IIIB and evidence of neurologic dysfunction in its Phase I/II non-randomized clinical trial of a one-time gene therapy with ABO-101. ABO-101 is Abeona’s designation for rAAV9.CMV.hNAGLU. The clinical trial is an open-label, dose-escalation trial of ABO-101 injected intravenously through a peripheral limb vein. Trial participants will receive a one-time intravenous injection of either high- or low-dose ABO-101. Researchers will then assess the treatment’s safety and effectiveness at six, 12, and 24 months after gene therapy administration. There are two clinic locations for this clinical trial: Nationwide Children’s Hospital in Columbus, Ohio, USA; and Hospital Clínico Universitario de Santiago, in Santiago De Compostela, Spain.