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 disorder that weakens the immune system, leaving patients vulnerable to frequent and life-threatening infections. Kresladi is a gene therapy that uses a modified virus to deliver a functional copy of the ITGB2 gene into a patient’s own blood stem cells. By restoring the missing protein needed for immune cell function, Kresladi may help patients fight infections more effectively.
