TransCode Therapeutics Inc. (NASDAQ: RNAZ)

TransCode Therapeutics Inc. (NASDAQ: RNAZ) is a clinical-stage biotechnology company developing RNA-based therapeutics intended to overcome the lethal challenges of metastatic cancer. By targeting the molecular underpinnings of metastasis with a novel delivery platform, TransCode aims to open new frontiers in cancer treatment. The company’s approach centers on overcoming one of the most persistent obstacles in RNA therapeutics: the efficient and precise delivery of these molecules to tumor sites within the human body.

At the heart of TransCode’s strategy is its proprietary TTX nanoparticle platform, designed to enable the systemic delivery of a broad range of nucleic acid payloads—including antisense oligonucleotides, RNAi, and CRISPR constructs—while minimizing immune response and off-target effects. This delivery engine is a foundational technology that can be applied across multiple cancer types and therapeutic modalities.

TransCode’s mission is to defeat metastatic disease by designing intelligent RNA therapeutics that can reach and neutralize previously inaccessible genetic targets.

The company is headquartered in Boston, Massachusetts.

Lead Candidate: TTX-MC138

The company’s lead therapeutic candidate, TTX-MC138, is a first-in-class RNA-based drug designed to inhibit microRNA-10b (miR-10b)—a genetic driver of metastatic progression that has been linked to poor patient outcomes in over 200 clinical studies. TTX-MC138 utilizes the TTX platform to systemically deliver an antisense oligonucleotide that binds to miR-10b, disrupting metastatic cell survival mechanisms.

Currently in a Phase I/II clinical trial, TTX-MC138 has shown evidence of pharmacodynamic activity and tumor site accumulation in early human studies, with no safety or allergic hypersensitivity concerns reported. Preclinical models have demonstrated complete regressions of metastatic disease in animals. The trial design includes a dose-escalation Phase 1a and a dose-expansion Phase 1b, evaluating safety, pharmacokinetics, and early tumor response in patients with advanced solid tumors.

Pipeline and Platform

Beyond TTX-MC138, TransCode is advancing a robust pipeline of RNA therapeutics that leverage its modular delivery platform. These include:

• TTX-siPDL1: An RNA interference candidate targeting immune checkpoint inhibition in pancreatic cancer (orphan drug designated).
• TTX-RIGA: A cancer-agnostic therapeutic designed to activate RIG-I, a cytosolic immune receptor that triggers tumor cell apoptosis.
• TTX-CRISPR: A CRISPR-based gene editing therapeutic aimed at precise genome modification for various tumor types.
• TTX-BEC (in partnership with Akribion Genomics): A novel CRISPR-based therapeutic using G-dase E, an engineered nuclease for cell killing.
• TTX-mRNA: An mRNA delivery candidate for tumor-targeted therapy.

Each candidate benefits from TransCode’s TTX delivery architecture, which enables tunable nanoparticle design, customizable coatings for enhanced tumor uptake, and smart-release capabilities. The platform’s versatility supports a range of payloads—from nucleic acids to proteins and radionuclides—positioning it as a backbone for future oncology innovations.

Market Opportunity

Metastatic cancer remains one of the most critical and underserved areas in global oncology. Approximately 90% of the 10 million cancer-related deaths worldwide each year are attributed to metastasis, rather than primary tumors. This reality underscores the urgent demand for therapies capable of preventing or reversing metastatic progression.

According to a report by Market Research Future, the global metastatic cancer treatment market is projected to grow from $96.52 billion in 2025 to $199.51 billion by 2034, representing a compound annual growth rate (CAGR) of 8.4% over the forecast period.

Despite major advances in immunotherapy and targeted small molecules, an estimated 80% of cancer-driving genetic targets remain out of reach for these modalities. RNA-based therapeutics offer a powerful solution—but only if their delivery can be precisely controlled. TransCode’s proprietary TTX platform directly addresses this challenge, potentially unlocking a vast new class of drugs and establishing the company as a differentiated player in a rapidly growing oncology market.

Leadership Team

Tom Fitzgerald, Interim CEO and Chief Financial Officer, brings over two decades of strategic and operational experience in life sciences. Before joining TransCode, he served as CFO at Velico Medical and had senior leadership roles at several early- and mid-stage medical technology companies. Fitzgerald’s background spans financial management, business operations, and strategic planning, making him well-suited to guide TransCode through its clinical development and capital formation activities.

Dr. Zdravka Medarova, Co-founder and Chief Scientific Officer, is the scientific innovator behind TransCode’s platform technology. A former faculty member at Harvard Medical School and senior researcher at Massachusetts General Hospital, Dr. Medarova has authored over 90 peer-reviewed publications and holds more than 20 patents in molecular imaging, oncology, and RNA therapeutics. Her expertise in cancer biology and targeted delivery forms the scientific backbone of the company’s therapeutic approach.‎