Marnetegragene Autotemcel: First Approval.

Abstract

Marnetegragene autotemcel (KRESLADI™), an autologous stem cell-based gene therapy that adds functional copies of the ITGB2 gene to haematopoietic stem cells, was developed by Rocket Pharmaceuticals for the treatment of leukocyte adhesion deficiency-I (LAD-I). In March 2026, marnetegragene autotemcel received its first approval in the USA for the treatment of paediatric patients with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic haematopoietic stem cell transplant. This article summarises the milestones in the development of marnetegragene autotemcel leading to this first approval for treatment of paediatric patients with severe LAD-I.