Trogenix Ltd has announced the publication of its breakthrough pre-clinical data in Nature reporting complete tumour eradication and durable protection in an aggressive brain cancer model that closely mimics human glioblastoma (GBM). The research supports an innovative approach to potentially achieving curative responses with long-term immunological protection against GBM, the most lethal form of brain cancer.
It underpins the company’s transition to a clinical-stage oncology company, with patient dosing in its first clinical trial for glioblastoma expected in Q2 2026.
The publication, authored by scientists from the University of Edinburgh, UCL Cancer Institute and The Royal Infirmary of Edinburgh, focusses on Trogenix’s synthetic super-enhancers (SSEs), which are engineered genetic constructs that act as selective transcriptional switches, designed to harness glioblastoma stem cells’ own unique transcriptional machinery. The SSEs, delivered using adeno-associated virus vectors (AAVs), specifically target SOX2 and SOX9-driven gene networks that are activated in patient glioblastoma stem cells.
Key findings of the study showed that, when delivered as a single treatment dose, SSEs led to: striking tumour regression within 1-2 weeks, and complete tumour clearance in 83% of treated cases over the subsequent 2-3 weeks; precision immune activation to eradicate tumours; no further tumour regrowth after the initial treatment, with no toxicity over the next 11 months; and no detectable tumour formation even after re-challenge.
Critically, SSE activity was validated using fresh patient GBM tissue samples, demonstrating selective expression in tumour cells but not in the healthy normal brain cells. This precision targeting of glioblastoma cells while sparing normal tissue minimises off-target toxicities and represents a key validation step before advancing to patient trials.
Steeve Pollard, study lead at the University of Edinburgh and chief scientific officer of Trogenix, said: “This pre-clinical work in an aggressive brain cancer model that closely mimics human glioblastoma has achieved what we thought impossible – complete tumour elimination and long-lasting protection against cancer recurrence without off-target toxicity using a single dose of a single agent. Our Synthetic Super-Enhancer technology combines the dual power of cancer cell killing and immune stimulation through a sophisticated ‘Trojan horse’ precision delivery method with the potential for transforming how we address GBM. We can finally hit the tumour hard and early by using controlled gene therapy that has been designed to be highly selective for the most aggressive cancer cells. We are committed to move these findings as quickly and safely as possible to patients and are optimistic that this can provide a new approach to tackling solid tumours. We look forward to starting our phase I/II ADePT trial for glioblastoma this year.”
The dual-payload approach delivers two therapeutic agents directly to cancer cells: HSV-TK for direct tumour killing (cytotoxic) and IL-12 for immune system activation (cytokine), creating synergistic effects that act as an in-situ vaccine. This approach generates durable immunological memory that prevents cancer recurrence by educating the immune system to detect cancer, transforming gene therapy from temporary intervention to lasting protection.
Iain Foulkes, chief executive of Cancer Research Horizons, said: “Around 3,200 people are diagnosed with glioblastoma every year in the UK, of which just 160 will survive for five years or more. That number is unacceptable and we urgently need better treatment options. This work lays the foundation for Trogenix’s next steps into early-stage clinical trials, steps that will hopefully take us closer to a world where fewer people lose their lives to brain cancer. Cancer Research Horizons remains committed to supporting Trogenix’s mission to target aggressive cancers, and we’re excited to see if their promising precision science can benefit patients.”