KRESLADI
KRESLADI (marnetegragene autotemcel) is a one-time ex vivo lentiviral gene therapy developed by Rocket Pharmaceuticals for leukocyte adhesion deficiency type I (LAD-I) — an ultra-rare, fatal pediatric immune disorder in which ITGB2 gene mutations prevent white blood cells from reaching sites of infection, leaving patients unable to fight bacteria. The therapy harvests a patient's own blood stem cells, inserts a functional ITGB2 gene via lentiviral vector, and reinfuses the corrected cells after myeloablative conditioning to restore immune function. Without treatment, severe LAD-I is typically fatal in infancy; KRESLADI received FDA Priority Review and would be Rocket's first commercial gene therapy.
Upcoming catalysts
Programs
Fanconi Anemia
Fanconi anemia is an ultra-rare inherited DNA repair disorder causing progressive aplastic anemia requiring transfusions, a high risk of AML, and associated developmental abnormalities; without treatment, most patients require hematopoietic stem cell transplantation with significant morbidity and limited donor availability. KRESLADI delivers a functional FANCA gene into the patient's own blood stem cells via lentiviral vector; it received FDA Priority Review and, if approved at the 2026 PDUFA, would be Rocket's first commercial gene therapy.