Abeona gains on early data for AAV9 MPS IIIB gene therapy

Abeona Therapeutics Inc. (NASDAQ:ABEO) was up $2.25 (14%) to $18.20 on Wednesday after reporting that its adeno-associated viral serotype 9 (AAV9)-based gene therapy ABO-101 was well tolerated with no treatment-related serious adverse events reported through day 30 in the first patient dosed in a Phase I/II trial to treat mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU) gene.

The patient received a single IV injection of 2x10^13 vector genomes per kg (vg/kg) ABO-101. Abeona also said ABO-101 significantly reduced cerebrospinal fluid (CSF), urine and plasma heparan sulfate levels and normalized NAGLU enzyme activity compared to baseline. The company plans to enroll three patients at the 2x10^13 vg/kg dose before escalating to a 5x10^13 vg/kg dose...