NIH R01 · 2024
Project Summary We propose a novel regulatory T (Treg) cell therapy to treat IPEX syndrome, a rare autoimmune monogenic disease. IPEX is a life-threatening disease caused by loss-of-function FOXP3 mutations leading to dysfunctional Treg cells. The only current curative treatment for IPEX is allogeneic hematopoietic stem cell transplantation (allo-HSCT), which is only available to a minority of patients. The proposed product, CD4LVFOXP3 consists of autologous CD4+ T cells that have undergone lentiviral vector (LV)-mediated gene transfer of wild-type human FOXP3 leading to persistent high FOXP3 expression and acquisition of Treg cell phenotype and function. CD4LVFOXP3 were granted Orphan Drug…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.