FDA Approves Gene Therapy for Severe Pediatric LAD-I

Approval expands treatment option for a rare pediatric immunodeficiency

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The FDA approved marnetegragene autotemcel (KRESLADI) for severe pediatric leukocyte adhesion deficiency-I without a matched donor.
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Severe leukocyte adhesion deficiency type I is a rare genetic disorder caused by ITGB2 gene variants, leading to severe infections.
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The incidence of severe LAD-I in the US is estimated at one in 100,000 to one in 200,000 live births, with high early childhood mortality.
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KRESLADI's approval is based on increased neutrophil CD18 and CD11a expression, with ongoing studies required for continued approval.
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The therapy's safety profile includes risks like serious infections and potential oncogenesis, necessitating long-term monitoring.

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