The U.S. FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel), marking the first gene therapy option for severe leukocyte adhesion deficiency-I (LAD-I). The decision clears Kresladi for pediatric patients with severe LAD-I due to biallelic variants in ITGB2 who do not have a matched sibling donor for allogeneic hematopoietic stem cell transplant. Rocket previously received an FDA rejection over manufacturing concerns, setting the stage for this regulatory turnaround. The approval arrives just ahead of the March 28 PDUFA date, per the reporting around the decision. For developers in ultra-rare immunology, the case underscores how manufacturing and product-consistency scrutiny remain pivotal, even when clinical need is high. It also reinforces FDA’s willingness to use accelerated pathways in small, high-unmet-need populations where traditional endpoints can be difficult to measure. The broader implication for the sector is that gene therapy pipelines will continue to face a tight linkage between manufacturing remediation and regulatory acceptance, especially for autologous approaches in pediatrics.