This work will translate recent advances in CRISPR-Cas9-based human T cell editing into generation of a novel gene-corrected regulatory T cell (gcTreg) therapy. These gcTregs will be developed initially for treatment of a family with a progressive autoimmune syndrome caused by mutations in the IL2RA gene. IL2RA is a surface protein important for the function of regulatory T cells (Tregs), a type of immune cell that plays a critical role in preventing autoimmunity and excessive inflammation. Patients with IL2RA mutations develop a progressive and often severe autoimmune syndrome.

We have developed a gene correction strategy for the most severely affected sibling in a family with IL2RA deficiency. Tregs will be isolated from this patient’s peripheral blood, gene-corrected, expanded ex vivo, and then reinfused within the context of a phase 1 clinical trial. We are currently in preclinical development and have initiated positive interactions with the FDA. A full Investigational New Drug (IND) application is planned in the near future.