TransCode Therapeutics Advances RNA Cancer Therapy, Reaches Milestone in Phase 1a Trial

TransCode Therapeutics has successfully dosed 15 patients in its Phase 1a trial for RNA-based cancer therapeutic TTX-MC138, with early data showing promising target engagement and no significant toxicities.

May 8, 2025
TransCode Therapeutics Advances RNA Cancer Therapy, Reaches Milestone in Phase 1a Trial

Biotech firm TransCode Therapeutics has reported significant progress in its RNA-based cancer therapy research, with 15 patients now treated across four dose levels in its Phase 1a clinical trial. The company's lead therapeutic candidate, TTX-MC138, targets metastatic tumors that overexpress microRNA-10b, a known biomarker of metastasis.

The Safety Review Committee has approved expanded enrollment after observing no significant toxicities or disease progression. Ten patients remain actively enrolled in the study, with the longest-treated patient demonstrating stable disease after seven treatment cycles. Early pharmacokinetic and pharmacodynamic data suggest promising target engagement, supporting the potential advancement to the Phase 1b dose expansion stage.

This development represents a critical milestone in developing innovative RNA therapeutics for cancer treatment. By focusing on a unique biomarker and utilizing a proprietary nanoparticle delivery platform, TransCode is addressing key challenges in RNA-based therapeutic approaches.

The trial's initial results suggest potential for a targeted treatment approach that could offer new hope for patients with metastatic cancers. The company's ability to demonstrate safety and initial efficacy at multiple dose levels indicates a promising path forward in developing novel cancer therapies.