FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I

United States, March 31 -- The U.S. Food and Drug Administration approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I). Kresladi is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. Severe LAD-I is a rare, inherited immune deficiency caused by mutations in the ITGB2 gene, which prevent white blood cells from effectively fighting infections. Patients with severe LAD-I experience recurrent, life-threatening bacterial and fungal infections with subs...