The FDA granted accelerated approval to Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel), positioning it as the first gene therapy option for severe leukocyte adhesion deficiency type I (LAD-I). The approval clears use for pediatric patients with severe LAD-I due to biallelic ITGB2 variants who lack an HLA-matched sibling donor for allogeneic hematopoietic stem cell transplant. The decision arrives after an earlier FDA refusal tied to manufacturing concerns in 2024, and it follows a marketing timeline that had Rocket preparing for a late-March PDUFA date. Commercial uptake is expected to be limited in scope given the disease’s ultrarare prevalence, but the approval meaningfully expands the regulatory path for gene therapies in immunodeficiency. Clinicians will now shift from donor-matched transplant strategies to a gene-therapy approach for eligible patients, potentially affecting how LAD-I treatment pathways are built for new diagnoses and referral networks.