TransCode Therapeutics (NASDAQ:RNAZ) today announced positive preclinical data demonstrating that its lead candidate, TTX-MC138, successfully reaches orthotopic glioblastoma tumors following intravenous administration and achieves sustained target engagement against miR-10b.
In murine models of glioblastoma, treatment with TTX-MC138 resulted in a 5-fold increase in apoptotic activity within tumors and delivered a statistically significant survival benefit compared to controls.
The company also confirmed that IND-enabling studies, including pharmacokinetics, biodistribution, and toxicology assessments, have been completed.
Phase 1 safety data are already available from non-CNS patients, providing additional support for advancing the program.
TransCode Therapeutics anticipates initiating a Phase 2a clinical trial in the first half of 2026.
Glioblastoma remains one of the most aggressive and difficult-to-treat brain cancers, and therapies capable of effectively crossing the blood-brain barrier while specifically targeting oncogenic drivers like miR-10b represent a critical area of unmet need.