marnetegragene autotemcel

Marnetegragene autotemcel (KRESLADI) is an autologous hematopoietic stem cell-based gene therapy developed by Rocket Pharmaceuticals for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I). The therapy utilizes a lentiviral vector to deliver a functional copy of the *ITGB2* gene into the patient's own CD34+ hematopoietic stem cells ex vivo. The *ITGB2* gene encodes for CD18, a critical component of beta-2 integrins required for leukocyte adhesion and migration to sites of infection. By restoring CD18 expression, the therapy enables the immune system to combat infections effectively. It was granted accelerated approval by the FDA in March 2026 for pediatric patients with severe LAD-I who lack an HLA-matched sibling donor.

Brand names

KRESLADI

Other names

Chim-CD18-WPREChim-CD-18-WPREChim-CD 18-WPRE