TransCode Therapeutics (RNAZ) reported that its lead RNA therapeutic, TTX‑MC138, achieved successful systemic delivery to glioblastoma multiforme (GBM) tumors in orthotopic mouse models, as published in the January 6 2026 issue of the Journal of Functional Biomaterials. The study, conducted in partnership with Michigan State University, showed that a single intravenous dose of TTX‑MC138 produced sustained target engagement within human GBM tumors, a five‑fold increase in apoptotic activity, and a statistically significant extension of survival in the animal cohort.
The data confirm that TransCode’s TTX platform can cross the blood‑brain barrier—a long‑standing obstacle for RNA therapeutics—providing a critical de‑risking step toward clinical evaluation of TTX‑MC138 in GBM patients. Dr. Zdravka Medarova, Chief Scientific Officer, noted that the findings “represent an important step forward in targeting one of the most treatment‑resistant forms of cancer” and that the platform’s delivery advantage could broaden its reach beyond metastatic solid tumors.
TransCode’s financial position remains a key context for the announcement. The company’s most recent quarterly report showed a Q3 2025 EPS of –$5.49, a miss relative to analysts’ expectations, reflecting ongoing cash burn and the need for additional capital. In October 2025, TransCode secured a $25 million investment—$20 million in cash and a $5 million note—to fund mid‑stage clinical trials, underscoring the importance of the preclinical milestone for future fundraising and partnership opportunities.
Market reaction to the preclinical data was positive, with the company’s stock surging 18 % on the day of the announcement. Investors viewed the successful delivery and therapeutic effect as a validation of the TTX platform’s core technology and a signal that the company is progressing toward the first clinical trial in GBM, which is planned for the first half of 2026. The surge also reflected confidence that the platform could be applied to other CNS indications, potentially expanding the company’s addressable market.
The preclinical success aligns with TransCode’s broader strategy of advancing TTX‑MC138 through IND‑enabling studies, PK/biodistribution, and toxicity work, and of initiating a Phase 1 safety study in non‑CNS patients. A Phase 2a trial in metastatic disease is anticipated in H1 2026, and the GBM data provide a strong rationale for expanding the clinical development program to a high‑need indication. The company’s leadership emphasized that the data “broadening the potential reach of our RNA‑based therapeutics beyond metastatic solid tumors” will be a key driver of future growth.
The announcement underscores TransCode’s progress in overcoming a critical delivery barrier, strengthens its pipeline, and positions the company for the next phase of clinical development while highlighting the financial challenges that necessitate continued capital raising. Investors will likely view the preclinical milestone as a positive step toward eventual regulatory approval and commercial viability, but will also monitor the company’s cash burn and funding strategy as it moves into clinical trials.
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