REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights
REGENXBIO reported fourth-quarter and full-year 2025 results and highlighted multiple near-term catalysts across its late-stage gene therapy portfolio for rare and retinal diseases. RGX-202 for Duchenne muscular dystrophy produced positive 18-month Phase I/II functional data and pivotal topline results are expected early Q2 2026, with enrollment and a planned pre-BLA interaction underway. Sura-vec (ABBV-RGX-314) has a wet AMD pivotal readout expected Q4 2026 and a Phase IIb/III diabetic retinopathy dosing milestone tied to a $100 million AbbVie payment expected Q2 2026. The FDA placed a clinical hold on RGX-111 following a reported intraventricular CNS tumor case; 2025 R&D spend rose to $228.3M and cash of $240.9M is expected to fund operations into early 2027.
Why it mattersFDA placed a clinical hold on RGX-111 after a CNS tumor case, raising regulatory risk and timing uncertainty for BLA plans.