REGENXBIO is a clinical-stage gene therapy company that develops one-time treatments using adeno-associated virus (AAV) vectors. The core premise is that a single gene therapy administration can replace defective genes and produce a lasting therapeutic effect, eliminating the need for chronic treatment. REGENXBIO has no approved products yet, but its pipeline is focused on three disease areas: retinal diseases, neuromuscular diseases, and neurodegenerative diseases. Its lead program, sura-vec (developed with AbbVie), is a one-time gene therapy for wet AMD and diabetic retinopathy that aims to replace lifelong anti-VEGF injections. Two pivotal trials with over 1,200 patients combined completed enrollment in late 2025, with topline data expected in Q4 2026. RGX-202 targets Duchenne muscular dystrophy and completed enrollment in a pivotal trial in late 2025, with topline data expected in early Q2 2026 and a BLA submission planned under accelerated approval. RGX-121 and RGX-111 target rare enzyme deficiency disorders but face near-term setbacks, including an FDA Complete Response Letter and a clinical hold. Alongside its pipeline, REGENXBIO licenses its NAV Technology Platform — a portfolio of over 100 AAV vectors — to other biopharma companies, generating royalties and milestone payments. The most significant license is Novartis' for Zolgensma and Itvisma, approved gene therapies for spinal muscular atrophy. REGENXBIO also owns and operates its own manufacturing facility in Rockville, Maryland, which it argues gives it control over clinical and commercial supply for its key programs.