Progress in RGX-202 Program for Duchenne
Enrollment completed ahead of schedule for the AFFINITY DUCHENNE pivotal trial. Strong safety and efficacy profile in Phase I/II with no serious adverse events reported. Top-line data expected in early Q2 2026, with a BLA submission planned for mid-2026.
Advancements in RGX-121 for MPS II
Positive 12-month data delivered to FDA, with no observations during site inspections. The program is on track for potential approval by February 2026.
Strong Financial Position
Cash, cash equivalents, and marketable securities of $302 million as of September 30, 2025. Cash runway expected to fund operations into early 2027, with potential nondilutive financing opportunities available.
Completion of Enrollment in Wet AMD Studies
Over 1,200 patients enrolled across 200 sites in the global Phase III studies for subretinal wet AMD, marking the largest global gene therapy program ever conducted.