Avrobio's Pompe Disease Gene Therapy Shows Efficacy, Safety In Preclinical Study
Avrobio Inc (NASDAQ:AVRO) announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in infantile-onset Pompe disease. Pompe disease is a lysosomal disorder caused by a mutation in the GAA gene. The lack of the enzyme encoded by GAA results in a toxic buildup of glycogen throughout the body and central nervous system.…
Benzinga ·
Benzinga
