Clinical Efficacy In DuchenneRGX-202 has shown sustained functional improvements and no serious adverse events in Duchenne patients, which bolsters the case for approval and market uptake if results hold in confirmatory studies.
Manufacturing CapabilityIn-house, end-to-end manufacturing with high purity metrics and fill-finish capabilities enables scalable commercial supply and can reduce cost of goods, supporting launch readiness for multiple gene therapy franchises.
Regulatory MilestonesPivotal AFFINITY topline results could support an accelerated biologics license application, creating a clear regulatory pathway for the Duchenne program and potential commercialization value.