Award
NIH Reporter #5R01FD007540-04
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
$697,971.00
Ceiling
$697,971.00
Awarded
August 07, 2026
Identifier
5R01FD007540-04
A Phase 1 clinical trial funded by the FDA to evaluate the safety, feasibility, and pharmacokinetics/pharmacodynamics of the gene therapy CD4LVFOXP3 for treating IPEX syndrome, conducted at Stanford University.
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 trial will also gather data on pharmacokinetics and pharmacodynamics of CD4LVFOXP3, which is instrumental for further clinical trials in IPEX and other autoimmune diseases.