The U.S. FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), marking the first gene therapy option for the ultrarare immune disorder. The approval comes ahead of the March 28 PDUFA date and covers pediatric patients with severe LAD-I due to biallelic variants in ITGB2 who lack a matched sibling donor. Rocket previously faced an FDA rejection in 2024 for manufacturing concerns, making Thursday’s approval a key reversal for the program. The therapy is designed as a one-time treatment intended to address the underlying immunodeficiency driving recurrent and fatal infections. Separately, the FDA also approved a Rocket gene therapy in a related report, reinforcing the approval’s placement as a rare-disease milestone. The decisions underscore continued FDA willingness to use accelerated pathways in genomically defined, high-unmet-need settings when manufacturing and clinical evidence align.