On March 26, 2026, the U.S. Food and Drug Administration approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel), the first gene therapy treatment for pediatric patients with severe Leukocyte Adhesion Deficiency Type I (LAD-I). LAD-I is an inherited immune deficiency cause by mutations in the ITGB2 gene that leaves patients susceptible to recurrent, life-threatening bacterial infections. Current treatment options consist of allogenic blood stem cell transplants, which according to the FDA “associated with significant morbidity and mortality, especially in patients without an HLA-matched sibling donor.” Kresladi consists of the patients own blood stem cells which isolated from the patient, genetically modified to introduce healthy copies of the ITGB2 gene, and then infused back into the patient to restore immune function.
According to the FDA, “[t]he safety and effectiveness of Kresladi were established in one open-label, single-arm, multicenter study based on [indicators of improved immune activity] at month 12 with sustained effect through month 24 post-infusion.” The application for Kresladi was granted multiple special designations including Orphan Drug, Rare Pediatric Disease, Regenerative Medicine Advanced Therapy and Fast Track designations. As a condition of Kresladi’s approval, Rocket Pharmaceuticals, Inc. will conduct post-approval studies to verify and describe the clinical benefits. Per the FDA, “[c]ontinued approval may be contingent upon verification of clinical benefit in confirmatory trials.”