The regulatory status of RP1 remains unsettled, however, as despite demonstrating clinically meaningful and durable responses in anti-PD-1-failed melanoma in the IGNYTE trial, the FDA has withheld approval pending confirmatory randomized data showing overall survival benefit, highlighting the ongoing debate over the sufficiency of single-arm response-based evidence for new therapies in this high-need population. The gene discussed is PDCD1; the disease is melanoma.