Australian researchers led by Dr. Esther Bandala-Sanchez, Dr. Miles Horton, and Dr. Tom Lew at WEHI have developed a novel prime editing gene-therapy platform that provides doctors with a control dial over cell therapies. Published in Blood Journal in October 2026, the strategy engineers therapeutic T cells to resist common immunosuppressants like tacrolimus while remaining sensitive to alternatives like cyclosporin, allowing edited cells to survive ongoing clinical immune suppression.
Development and publication of research
- ▪The WEHI-led research on drug-controllable prime-edited T-cell therapies was published in Blood Journal in October 2026.
- ▪WEHI researchers Dr. Esther Bandala-Sanchez, Dr. Miles Horton, and Dr. Tom Lew developed a prime editing gene-therapy platform to give doctors precise control over cell therapies.
Mechanism of drug-resistant cell editing
- ▪Using prime editing, researchers rewired T cells to make them resistant to the immunosuppressive drug tacrolimus while keeping them sensitive to cyclosporin.
- ▪The prime editing platform allows clinicians to suppress a patient's broader immune system while preserving edited therapeutic cells that survive under ongoing immunosuppression.
Use of existing approved drugs
- ▪Unlike competing approaches requiring approval of new cellular products and novel drug molecules simultaneously, WEHI's platform relies on existing FDA-approved immunosuppressants.
- ▪The research team designed the gene-editing platform around existing hospital pharmacy drugs like tacrolimus and cyclosporin to streamline translation into routine patient care.
Testing in specific cells and diseases
- ▪The WEHI-developed prime editing platform was successfully evaluated in virus-specific T cells for transplant recipients and CAR T cells used in cancer treatment.
- ▪Researchers used prime editing on cells from patients with SPTCL-HLH, a rare blood cancer affecting children and young adults, to correct a disease-causing mutation and confer tacrolimus resistance.
Advancement toward clinical trials
- ▪The planned clinical trial aims to make controllable cell therapies feasible for organ and stem cell transplant recipients, rare immune disorder patients, and cancer patients requiring ongoing immunosuppression.
- ▪Following successful preclinical T-cell engineering, the WEHI-led research team is advancing the prime editing platform toward its first-in-human clinical trial.
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