Abeona Therapeutics Reports on Initial Safety and Biopotency Signals...

First-in-man AAV9-based gene therapy delivered by single intravenous injection targeting central nervous system and peripheral manifestations of MPS IIIB. ABO-101 is well tolerated and demonstrates early biopotency signals with significant disease-specific heparan sulfate reductions in cerebral spinal fluid, urine, and plasma and greater than 300-fold increase in NAGLU enzyme activity observed in first subject at 30 days post injection.