Award
Food and Drug Administration 5R01FD007540-03
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome [revised IND and clinical protocol to be submitted to FDA by 10/11/2021]
Recipient
Stanford University, Department of Pediatrics
Award Amount
$669,615.00
Ceiling
$669,615.00
Awarded
August 02, 2024
Identifier
5R01FD007540-03
This award funds a Phase 1 clinical trial at Stanford University to develop and test a novel gene therapy for IPEX syndrome, aiming to improve safety and efficacy of treatment for this rare autoimmune disease.
Description
A Phase 1 clinical trial to test the feasibility of manufacturing and safety of administering autologous CD4+ T cells converted into CD4LVFOXP3 Treg-like cells by lentiviral vector-mediated FOXP3 gene transfer in participants with IPEX syndrome, a rare autoimmune monogenic disease. The trial aims to reduce immune dysregulation and autoimmune manifestations, evaluate clinical impact, and explore pharmacokinetics and pharmacodynamics. The project addresses a significant unmet medical need and has potential broader application to other autoimmune diseases.