20 September 2022 - AVR-RD-04 has previously received orphan drug designation from FDA and EMA.
AvroBio today announced that the US FDA has granted rare paediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis, a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease.