Trogenix's Gene Therapy Achieves 83% Complete Tumor Elimination in Glioblastoma Model, Nature Study Shows
Key Insights
Trogenix (search)'s Synthetic Super-Enhancers (search) achieved complete tumor elimination in 83% of treated glioblastoma (search) cases with a single dose and no toxicity over 11 months.
The dual-payload gene therapy platform combines direct tumor killing with immune activation, creating durable protection against cancer recurrence.
The breakthrough pre-clinical data published in Nature supports the company's transition to clinical trials, with patient dosing expected in Q2 2026.
Trogenix (search) Ltd has published breakthrough pre-clinical data in Nature demonstrating that its proprietary gene therapy platform achieved complete tumor elimination in 83% of treated cases in an aggressive brain cancer (search) model that closely mimics human glioblastoma (search). The single-dose treatment showed no toxicity over 11 months and prevented tumor recurrence, marking a significant advancement in the fight against the most lethal form of brain cancer.
Revolutionary Gene Therapy Platform Shows Unprecedented Results
The study, conducted by scientists from the University of Edinburgh, UCL Cancer Institute, and The Royal Infirmary of Edinburgh, focused on Trogenix (search)'s Synthetic Super-Enhancers (search) (SSEs) - engineered genetic constructs that act as selective transcriptional switches. These SSEs are delivered using adeno-associated virus vectors (AAVs) and specifically target SOX2 (search) and SOX9 (search)-driven gene networks activated in patient glioblastoma (search) stem cells.
The key findings revealed striking tumor regression within 1-2 weeks, with complete tumor clearance in 83% of treated cases over the subsequent 2-3 weeks. Critically, the treatment showed precision immune activation to eradicate tumors, no further tumor regrowth after initial treatment, and no detectable tumor formation even after re-challenge.
Dual-Payload Approach Creates Lasting Protection
The innovative dual-payload approach delivers two therapeutic agents directly to cancer cells: HSV-TK for direct tumor killing (cytotoxic) and IL-12 (search) for immune system activation (cytokine). This combination creates synergistic effects that act as an in-situ vaccine, generating durable immunological memory that prevents cancer recurrence by educating the immune system to detect cancer.
Professor Steve Pollard, Study Lead at the University of Edinburgh and Chief Scientific Officer of Trogenix (search), explained: "This pre-clinical work in an aggressive brain cancer (search) model that closely mimics human glioblastoma (search) 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."
Precision Targeting Validated in Patient Tissue
A critical validation step involved testing SSE activity using fresh patient glioblastoma (search) tissue samples, which demonstrated selective expression in tumor cells but not in healthy normal brain cells. This precision targeting of glioblastoma cells while sparing normal tissue minimizes off-target toxicities and represents a key milestone before advancing to patient trials.
Addressing Critical Medical Need
The research addresses a devastating medical need, as approximately 3,200 people are diagnosed with glioblastoma (search) every year in the UK, with only 160 surviving for five years or more. Dr Iain Foulkes, Chief Executive of Cancer Research Horizons, emphasized: "That number is unacceptable and we urgently need better treatment options. This work lays the foundation for Trogenix (search)'s next steps into early-stage clinical trials."
Clinical Translation Timeline
The published pre-clinical data underpins Trogenix (search)'s transition to a clinical-stage oncology company, with patient dosing in its first clinical trial for glioblastoma (search) expected in Q2 2026. Professor Pollard noted: "We look forward to starting our Phase I/II ADePT trial for glioblastoma this year."
The research was funded by Cancer Research UK and the Biotechnology and Biological Sciences Research Council (BBSRC). Trogenix (search), which spun out of the University of Edinburgh in 2024, completed a £70m/$95m Series A financing in October 2025, led by IQ Capital with participation from existing investors and new investors including Eli Lilly & Co.