The FDA has approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel) as the first gene therapy for severe leukocyte adhesion deficiency type I (LAD-I), granting accelerated approval for pediatric patients without a matched sibling donor. The decision clears the path for a one-time treatment in an ultra-rare immunodeficiency associated with recurrent, often fatal infections. The approval follows an earlier FDA rejection related to manufacturing issues, making this the second major checkpoint for Rocket’s platform and rare-disease regulatory strategy. Kresladi was cleared ahead of its PDUFA date, underscoring the agency’s willingness to move quickly in high-unmet-need settings. Separately, the agency also greenlit Denali Therapeutics’ Avlayah for Hunter syndrome using Denali’s brain-penetrant delivery technology to cross the blood-brain barrier. The approvals together highlight continued FDA momentum in delivery-engineered rare disease therapies and gene therapy.