ARTICLE | Clinical News

Abeona reports early data for AAV9 MPS IIIB gene therapy

February 9, 2018 7:39 PM UTC

Abeona Therapeutics Inc. (NASDAQ:ABEO) reported 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 type B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU) gene.

The patient received a single IV injection of 2x1013 vector genomes per kg (vg/kg) ABO-101. Abeona also said ABO-101 significantly reduced heparin sulfate levels in the cerebrospinal fluid (CSF) by 50%, urine by 69% and plasma by 60%. ABO-101 also reduced urinary total glycosaminoglycans (GAGs) by 67% and normalized NAGLU enzyme activity compared to baseline. The company plans to enroll three patients at the 2x1013 vg/kg dose before escalating to a 5x1013 vg/kg dose...

BCIQ Company Profiles

Abeona Therapeutics Inc.