Regenxbio reported that its Duchenne muscular dystrophy gene therapy met a pivotal clinical milestone, aiming the program toward an FDA submission. The company said the therapy achieved sufficiently high levels of a miniaturized muscle protein in the trial, clearing a key bar in its development plan. The update comes as the field reassesses safety and efficacy thresholds for DMD gene transfer following earlier concerns around tolerability. Regenxbio’s CEO framed the data as supporting a path for accelerated review, while timelines are now tied to FDA feedback. If the submission proceeds as planned, it would further validate the gene therapy strategy for DMD and intensify competition among dystrophy programs entering late-stage development.