US FDA grants first gene therapy for ultra-rare immune deficiency

The FDA granted accelerated approval to Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel), the first gene therapy option for severe leukocyte adhesion deficiency-I (LAD-I). The approval covers pediatric patients with severe LAD-I due to biallelic ITGB2 variants who lack an available HLA-matched sibling donor for transplant.