marnetegene autotemcel

LADICell (marnetegene autotemcel) is an investigational ex vivo lentiviral vector-based gene therapy developed by Rocket Pharmaceuticals for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare and often fatal pediatric genetic disorder caused by mutations in the ITGB2 gene, which encodes the CD18 subunit of the beta-2 integrin family. This deficiency impairs the ability of white blood cells to adhere to the endothelium and migrate to sites of infection, resulting in recurrent, life-threatening infections. LADICell utilizes a lentiviral vector to deliver a functional copy of the ITGB2 gene into the patient's own hematopoietic stem cells. Once re-infused, these modified cells differentiate into functional leukocytes that express CD18, thereby restoring the immune system's ability to combat infections. The therapy has received Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Orphan Drug designations from the FDA.

Brand names

LADICell

Other names

marnetegene autotemcelRP-L201RP-L-201RP-L 201