Posted by Michael Wonder on 23 Apr 2018
Abeona announces FDA grants RMAT designation to ABO-102 gene therapy in MPS IIIA
23 April 2018 - First gene therapy using AAV approach granted regenerative medicine advanced therapy designation.
Abeona Therapeutics announced today that the US FDA has granted regenerative medicine advanced therapy designation to ABO-102, the Company’s AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.
The Company continues to engage the FDA on its ongoing Phase 1/2 trial. In the trial, subjects receive a single intravenous injection of ABO-102 to facilitate systemic delivery of a functional copy of the gene associated with onset and progression of MPS IIIA. Subjects are evaluated at multiple time points post-injection for safety, tolerability and efficacy.
