The FDA cleared Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), naming it the first gene therapy option for this ultrarare immunodeficiency. The accelerated approval supports pediatric patients with severe LAD-I due to biallelic ITGB2 variants who lack an HLA-matched sibling donor for allogeneic hematopoietic stem cell transplant. Separately, a second FDA approval item in the feed reiterates the same accelerated approval decision timeline and patient eligibility details. Together, the disclosures underscore the regulator’s willingness to advance gene therapies in settings with limited treatment options and urgent unmet need.