for severe leukocyte adhesion deficiency type I (LAD-I)
BLA· Priority Review · Orphan · RMAT
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).
About
Severe leukocyte adhesion deficiency type I (LAD-I) is a rare genetic immune disorder where white blood cells cannot travel to sites of infection, leading to life-threatening recurrent infections. Kresladi is a one-time gene therapy that uses a patient's own modified cells to deliver a functional copy of the ITGB2 gene. This aims to restore the white blood cells' ability to fight infections, potentially reducing the severe complications of the disease.
