Model LAD-I -Leukocyte Adhesion Deficiency
LAD-I is a rare genetic disorder affecting the immune system and is caused by a defect in the ITGB2 gene, leading to a deficiency in CD18. Due to this deficiency, white blood cells (primarily neutrophils) are unable to leave the bloodstream to go to the site of infection. As a result, patients with severe LAD-I can develop life-threatening infections which they are unable to fight. Without a successful bone marrow transplant, severe LAD-I is frequently fatal during the first 2 years of life.
RP-L201 is a gene therapy product containing autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to contain a functional copy of the ITGB2 gene. In an early-stage clinical trial, RP-L201 has been shown to increase levels of CD18, correcting the deficiency that is a hallmark of the disease.