New compelling clinical data from first-ever Gaucher disease type 3 (GD3) patient and four Gaucher disease type 1 (GD1) patients dosed with investigational AVR-RD-02
Data from first pediatric GD3 patient, the more
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease.