REGENXBIO receives FDA response for RGX-121 application
The FDA accepted the gene therapy’s application under the accelerated approval pathway in May 2025. The CRL indicated that the FDA agreed in principle with the study protocol
Denali’s lead investigational TransportVehicle-enabled enzyme replacement therapy is intended to treat mucopolysaccharidosis type II (Hunter syndrome, a rare genetic disorder affecting boys). The US Food and Drug Administration