NHS BLOOD AND TRANSPLANT

Pioneering CRISPR Gene Therapy for CTLA-4 Insufficiency Advances Toward First-in-Human Trial

- UCL scientists are developing a first-of-its-kind gene therapy for CTLA-4 insufficiency, a rare inherited immune disorder with limited treatment options. - The approach uses CRISPR/Cas9 to replace the faulty CTLA-4 gene in patients' own T cells, addressing the root cause rather than managing symptoms. - Early pre-clinical studies show corrected cells lead to better immune regulation, with a Phase I trial in up to 8 patients planned for 2028. - The collaboration involves UCL, NHS Blood and Transplant, Great Ormond Street Hospital, and is funded by medical research organization LifeArc.