Rocket Pharmaceuticals said the FDA approved its gene therapy Kresladi (marnetegragene autotemcel) for leukocyte adhesion deficiency type I, and the program earned a rare pediatric disease priority review voucher. The company framed the decision as the first BLA to pass CBER this year, and said it plans to launch Kresladi by year-end. The approval also highlights Rocket’s commercial readiness after CBER review—an important step for gene therapies aiming to scale beyond development. The use of a priority review voucher underscores the regulator’s focus on pediatric targeting and accelerated timelines for rare diseases. While details on launch execution weren’t expanded, the path to broader adoption will hinge on manufacturing scale-up, payer alignment, and post-market evidence generation—key execution areas for one-and-done gene therapies.