Posted by Michael Wonder on 03 Nov 2021
AvroBio receives rare paediatric disease designation from the U.S. FDA for AVR-RD-05, a gene therapy for mucopolysaccharidosis type II or Hunter syndrome
3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its lentiviral gene therapy for the treatment of mucopolysaccharidosis type II, or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.
The company’s planned investigator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in the second half of 2022.
