Award
NIH Reporter #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
Award Amount
$669,615.00
Ceiling
$669,615.00
Awarded
August 02, 2024
Identifier
5R01FD007540-03
A Phase 1 clinical trial to evaluate the safety and feasibility of a gene therapy for IPEX syndrome using autologous Treg-like cells generated via lentiviral vector-mediated FOXP3 gene transfer.
Description
This project involves a Phase 1 clinical trial to test the safety and feasibility of an innovative gene therapy, CD4LVFOXP3, aimed at providing functional autologous Treg-like cells as a treatment for patients with IPEX syndrome. The therapy involves lentiviral vector-mediated gene transfer of FOXP3 into autologous CD4+ T cells, which are then used to reduce immune dysregulation and autoimmune manifestations. The trial will also gather pharmacokinetics and pharmacodynamics data to support further clinical development.