Regenxbio said its Duchenne muscular dystrophy gene therapy met the bar in a pivotal study, setting up an FDA submission expected in 2027. The company reported the therapy produced sufficiently high levels of a key miniaturized muscle protein broken in Duchenne. The milestone comes after safety scrutiny across the field for Duchenne gene therapies, including heightened attention following deaths associated with liver failure in prior programs. Regenxbio positioned its data as supportive of accelerated approval concepts. Separately, Regenxbio CEO Curran Simpson told STAT the dataset is designed to “check every single box” for accelerated approval, while framing the results as an effort to deliver a safer and more effective alternative to existing therapies. If accepted by regulators, Regenxbio’s submission would add another gene-therapy option for a market that has moved quickly from early-stage promise to post-approval safety expectations.