5 Key Takeaways

• 1

  The FDA approved marnetegragene autotemcel (KRESLADI) for severe pediatric leukocyte adhesion deficiency-I without a matched donor.

• 2

  Severe leukocyte adhesion deficiency type I is a rare genetic disorder caused by ITGB2 gene variants, leading to severe infections.

• 3

  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.

• 4

  KRESLADI's approval is based on increased neutrophil CD18 and CD11a expression, with ongoing studies required for continued approval.

• 5

  The therapy's safety profile includes risks like serious infections and potential oncogenesis, necessitating long-term monitoring.