FDA approved Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for leukocyte adhesion deficiency type I, according to the company’s March update. Rocket said the lentiviral-vector therapy, designed to deliver MAC-1, is expected to launch by year-end. The approval included a rare pediatric disease priority review voucher (PRV), Rocket said—an element investors will watch as more gene therapies move through CBER/CBER review pathways. Rocket also described Kresladi as the first BLA to pass through CBER this year. For gene-therapy developers, the event signals continued regulatory momentum for pediatric-focused, cell/gene products—especially those built on repeatable manufacturing and clear clinical endpoints in rare disease settings.