The FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency type I (LAD-I), marking the first gene therapy option for the ultrarare immune disorder. The approval clears Kresladi for pediatric patients with severe LAD-I caused by biallelic ITGB2 variants who lack an HLA-matched sibling donor for allogeneic hematopoietic stem cell transplant. The decision follows an earlier FDA refusal related to manufacturing issues in 2024, setting up a regulatory turnaround for Rocket’s autologous cell-based approach. The article notes an approval timing just ahead of a March 28 PDUFA date, underscoring the FDA’s continuing reliance on accelerated pathways for small, high-unmet-need populations. For investors and clinical stakeholders, the approval is a concrete data point in the rare-disease gene therapy pipeline and may influence expectations for other manufacturing-sensitive programs seeking FDA reconsideration.