BOSTON, Mass. — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA therapeutics for the treatment of high risk and advanced cancer, today announced the publication of preclinical research supporting the application of its lead candidate, TTX-MC138, for the treatment of glioblastoma multiforme (GBM). The article was published in the peer-reviewed Journal of Functional Biomaterials (Volume 17, Issue 1). The study, entitled “Nanotherapy Targeting miR-10b Improves Survival in Orthotopic Glioblastoma Models,” resulted from a collaboration between TransCode and Michigan State University. The study was led by Dr. Anna Moore, Professor, Director of the Precision Health Program, and Associate Dean for Research Development at the College of Human Medicine at Michigan State University and scientific co-founder of TransCode.
Glioblastoma is the most aggressive primary brain cancer, with median survival under two years from diagnosis despite current standard-of-care interventions. The molecular target of TTX-MC138, microRNA10-b (miR-10b), is highly expressed in GBM cells where it drives tumor survival, growth and invasiveness.
The study demonstrated delivery of TTX-MC138 to human GBM tumors implanted into the brains of murine models after intravenous injection, resulting in sustained target engagement within the tumor. TTX-MC138 also induced apoptotic activity in tumors by five-fold, consistent with observed induction of tumor cell death. Importantly, treatment with TTX-MC138 resulted in a statistically significant increase in survival.
These findings demonstrate the capability of TransCode’s TTX platform to systemically deliver antisense oligonucleotides (ASOs) to brain neoplasms and further supports its potential utility in overcoming key delivery barriers, including nucleic acid degradation and limited tumor penetration. Considering that investigational new drug (IND) enabling studies as well as pharmacokinetics, biodistribution, and required toxicity studies for TTX-MC138 have already been completed and that the formulation has shown appreciable safety in Phase I clinical trials in patients with non-central nervous system (CNS) cancers, these results support advancing TTX-MC138 to future clinical evaluation in patients with GBM.
“This research represents an important step forward in targeting one of the most treatment-resistant forms of cancer,” said Dr. Zdravka Medarova, CSO of TransCode. “By pairing our differentiated delivery approach with robust biological support, we are broadening the potential reach of our RNA-based therapeutics beyond metastatic solid tumors,” added Dr. Medarova.
TTX-MC138 is currently evaluated in metastatic disease in a Phase 1a clinical trial, with a Phase 2a clinical trial anticipated to begin in the first half of 2026, underscoring the translational relevance of this approach.
About TransCode Therapeutics
TransCode Therapeutics is a clinical stage company pioneering immuno-oncology and RNA therapeutic treatments of high risk and advanced cancers. The company’s lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors that overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class therapeutic candidates designed to mobilize the immune system to recognize and destroy cancer cells. For more information, visit www.transcodetherapeutics.com.
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