for severe leukocyte adhesion deficiency type I (LAD-I)
FDA approved Kresladi at/near the April 14, 2026 PDUFA date — the first gene therapy approved for severe LAD-I and a platform validation for Rocket's lentiviral technology. Sponsor monetized the resulting Priority Review Voucher for $180M shortly after approval (April 28, 2026).
Severe Leukocyte Adhesion Deficiency Type I (LAD-I) is an inherited immune disorder where white blood cells cannot exit the bloodstream and travel to sites of infection, leading to recurrent, life-threatening infections. Kresladi (marnetegragene autotemcel) is a one-time gene therapy that delivers a corrected copy of the essential ITGB2 gene into a patient's own blood stem cells. This treatment could restore the ability of white blood cells to fight infection, potentially preventing the severe complications that characterize the disease.