BioCentury
ARTICLE | Clinical News

Abeona reports updated data for MPS IIIA gene therapy

May 18, 2018 11:26 PM UTC

Abeona Therapeutics Inc. (NASDAQ:ABEO) said ABO-102 led to significant dose- and time-dependent reductions in CSF and urine heparan sulfate levels through 18 months in a Phase I/II trial to treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome). Data were presented at the American Society of Gene and Cell Therapy (ASGCT) meeting in Chicago.

ABO-102 is an adeno-associated viral serotype 9 (AAV9) vector that delivers the N-sulfoglucosamine sulfohydrolase (SGSH; HNS) gene...