FDA Approves Gene Therapy for Severe Pediatric LAD-I

Approval expands treatment option for a rare pediatric immunodeficiency

By
Kathryn Wighton
March 27, 2026
2 min

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The US FDA has granted accelerated approval for marnetegragene autotemcel (KRESLADI) to treat pediatric patients with severe leukocyte adhesion deficiency-I caused by biallelic variants in ITGB2. This condition, characterized by recurrent infections in infants due to impaired leukocyte adhesion, has a high early childhood mortality rate without treatment. The therapy is approved for children lacking a matched sibling donor for stem cell transplant, with benefits demonstrated through increased neutrophil counts. Ongoing monitoring is required for safety and efficacy.