Abeona Therapeutics Releases Updated Data On ABO-102 Therapy

Abeona Therapeutics released updated data from its ongoing clinical trial of its ABO-102, an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome Type A, at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. The ongoing phase 1/2 trial for ABO-102 is a first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for subjects with Sanfilippo syndrome (MPSIIIA), a rare autosomal-recessive lysosomal storage disease.

The clinical trial, which has received Fast Track designation, Orphan Product Designation, and Rare Pediatric Disease designation by the U.S. Food and Drug Administration, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2,” said Timothy J. Miller, Ph.D., president and chief executive of Abeona. “We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year.”

By Jon Flanagan