FOXP3-T4

FOXP3-T4 is an autologous gene-modified cell therapy developed by Assistance Publique - Hôpitaux de Paris (AP-HP) for the treatment of IPEX syndrome (Immune dysregulation, Polyendocrinopathy, Enteropathy, X-linked). The therapy involves the isolation of autologous CD4+ T cells, which are then transduced with a lentiviral vector to constitutively express the FOXP3 transcription factor. This process effectively converts effector T cells into functional regulatory T cells (Tregs). IPEX syndrome is caused by mutations in the FOXP3 gene, leading to a deficiency in Tregs and subsequent multi-organ autoimmunity. By restoring FOXP3 expression, FOXP3-T4 aims to re-establish immune tolerance. It is currently being evaluated in the Phase I/II THERIPEX clinical trial, potentially in combination with low-dose interleukin-2 (IL-2) to support cell survival and function.

Other names

THERIPEX