Regenxbio reported a clean safety profile and sustained functional gains from its RGX‑202 gene therapy in Duchenne muscular dystrophy (DMD) as the program approaches a pivotal data readout. Interim phase I/II Affinity trial results presented at the MDA conference showed functional improvements, stable cardiac assessments and tolerability consistent with prior cohorts. Company statements and conference disclosures indicate the dataset remains supportive heading into the next regulatory stage, though investors note the broader DMD gene‑therapy environment is under intense scrutiny following recent regulatory and safety debates in the space. Regenxbio said it is continuing to monitor endpoints and safety signals as dosing and follow‑up mature. Clinicians and payors will be watching the upcoming pivotal readout for efficacy durability, manufacturing consistency and safety markers that could affect labeling and coverage. The program’s outcome will influence competitive positioning across the DMD gene‑therapy field.