| Adding to the momentum, the FDA has granted Orphan Designation for pancreatic cancer, one of the deadliest and most challenging types to treat.
This signals a strong degree of regulatory alignment and highlights the clinical significance of their target, miRNA-10b, which has been closely linked to the spread and progression of multiple aggressive cancers.
But this isn’t a single-shot pipeline.
TransCode Therapeutics (Nasdaq: RNAZ)’s delivery system has broad potential across multiple RNA modalities, capable of transporting siRNA, antisense oligos, and immunostimulatory payloads with high precision.
The company is already expanding the platform with new candidates—TTX-siPDL1, a checkpoint inhibitor designed to improve immune engagement, and TTX-RIGA, which aims to activate the body’s innate immune response to help eliminate tumors from within.
In Preclinical Models—Complete Regressions…
Backing this clinical ambition is animal data that’s hard to overlook. In both murine models and spontaneous feline cancers, TTX-MC138 has delivered complete regressions of metastatic disease and extended survival significantly—even in late-stage cases.
These results speak to the potential power of the platform when it translates from lab to clinic.
One Platform. Multiple Targets. (RNAZ) Quietly Builds a Therapeutic Arsenal…
On March 27, 2025, TransCode announced that it had initiated dosing in its fourth cohort of the ongoing Phase 1 trial for TTX-MC138. This marks a key step forward in the program, with ten patients now treated at escalating dose levels.
The company’s ability to progress this trial without interruption adds meaningful weight to its early-stage development and reinforces that momentum is not just theoretical—it’s being measured one patient at a time.
Now that they’ve proven the delivery system can reach the hardest-to-hit areas—metastatic sites—the company is rolling out new candidates using the same backbone.
Their siRNA-based checkpoint inhibitor (TTX-siPDL1) has already shown potential to regress pancreatic tumors by ~90% in animal models when combined with chemo. Another, TTX-RIGA, is being positioned as an immune trigger with possible impact across recurrent or deep-tissue tumors.
The pipeline is no longer just one bullet.
It’s a modular platform—one with the potential to address multiple angles of cancer treatment using custom-built RNA tools.
And right now, that platform is finally stepping out of the shadows and into the spotlight.
With key milestones stacking up—and the clinical picture coming into sharper focus—there’s real reason to be paying attention. | 
