FDA Clears First Gene Therapy for Severe LAD-I in Children
The FDA approved Kresladi (marnetegragene autotemcel), the first gene therapy for severe Leukocyte Adhesion Deficiency Type I in pediatric patients without an HLA-matched donor, using autologous gene-modified stem cells to restore ITGB2 function and CD18/CD11a on white blood cells. The treatment is given as a single IV infusion after conditioning, based on biomarker improvements in an open-label trial, with post-marketing confirmatory studies required and safety monitoring for cytopenias and infections.