Patients & Caregivers:
Leukocyte Adhesion Deficiency-I (LAD-I)
Resources

LAD-I is a rare genetic immune disorder that affects white blood cells’ ability to reach sites of infections or injury to fight off pathogens, causing the immune system to malfunction. Ultimately, the condition prevents a patient’s immune system from adequately responding to infection and injury, predisposing patients to recurrent and often fatal infections. Children born with severe LAD-I begin experiencing recurrent and life-threatening infections immediately after birth and without a successful bone marrow transplant, survival beyond childhood is rare.
RP-L201 is an investigational gene therapy product containing autologous or patient-derived hematopoietic stem cells (HSCs) that have been modified with a lentiviral vector to contain a functional copy of the gene responsible for LAD-I, ITGB2. RP-L201 has the potential to restore immune function, avoiding the onset of severe, recurrent life-threatening infections.
For more information, contact [email protected].