Clinical Research & Medicines

UNLOCKING THE NEXT GENERATION OF CANCER-FIGHTING TREATMENTS

June 09, 2025

Regeneron’s proprietary VelociVax™ platform enables development and validation of potential new mRNA-based immunotherapies.

By: Johanna Hansen, PhD, Senior Director of Vaccine Technology

Messenger RNA (mRNA) is the biological blueprint that instructs cells to produce proteins—the essential building blocks that power nearly every process in the body. It stands for messenger ribonucleic acid and is a molecule that carries genetic information from DNA to the part of a cell that makes proteins. This capacity to swiftly deliver precise instructions has made mRNA a groundbreaking tool in drug development, enabling rapid design and manufacturing of targeted therapies. From enhancing cancer treatments to combating infectious diseases, mRNA’s adaptability is redefining what’s possible in medicine. For cancer therapeutics, the aim is to design mRNA to match the genetic signature of a patient’s tumor, enabling a more personalized treatment that could potentially result in a positive reaction from the body’s immune system.

Building on this transformative potential, we developed VelociVax™—a novel mRNA-backbone technology that provides a flexible foundation for designing and producing targeted proteins, and the latest technology to join our VelociSuite® technology platform. By enabling more agile combination testing and accelerating preclinical research, VelociVax sets the stage for potential breakthroughs in immune-modulating therapies.

Headshot of Johanna Hansen

Johanna Hansen,
PhD, Senior Director of Vaccine Technology

Strengthening Our In-House mRNA Capabilities

At Regeneron, we have long recognized the immense potential of tapping into genetics to address human health. While external collaborations remain an important part of our research ecosystem, we also saw an opportunity to accelerate our work by building an in-house platform. By doing so, we could design, produce, and validate mRNA constructs more rapidly, enabling our teams to pivot quickly based on emerging data and tailor our efforts to a wide range of research programs. This vision ultimately led to the creation of VelociVax, an internal platform that strengthens our capabilities and positions us to explore the full promise of mRNA technology. When you hear “vax,” people often think of preventative vaccines, however, this platform enables us to develop potential treatments. We use the term “vax” or “vaccine” here because, like preventative vaccines, the investigational treatments produced by our platform stimulate the body’s own immune system to fight cells with abnormal growth – in this case, cancer cells that are already present in the body. 

At its core, VelociVax focuses on generating mRNA constructs that encode key antigens, cytokines, or antibody fragments to spark or strengthen an immune response. The term “mRNA” may be familiar because of its recent use in infectious disease vaccines, but in fact this naturally-occurring biological component has been used for decades in treatments for multiple conditions. Using our standardized mRNA backbone—which serves as a foundational template our scientists can quickly customize to express proteins that stimulate a beneficial immune response—we have achieved greater flexibility and efficiency.

But, this is not just about faster turnaround; it’s about reimagining how we develop and optimize immunotherapies. Our platform empowers us to test multiple iterations in parallel, thereby identifying the most potent lead for further development.

VelociVax also offers the flexibility to combine mRNA-based agents with other potential strategies within our oncology portfolio—such as checkpoint inhibitors and bispecific antibodies—enabling rapid evaluation of potential synergies. By seamlessly layering new mRNA constructs onto other immunotherapy regimens, we can more swiftly pinpoint combinations with the greatest promise.

Advancing Research & Preclinical Development

VelociVax’s strength lies in its end-to-end approach. From the moment a gene of interest is identified—be it a tumor neoantigen uncovered by advanced sequencing techniques or an immune modulator that could enhance T cell activation—the process begins with cloning this sequence into our unique mRNA backbone. The subsequent in-house in vitro transcription reaction transforms the DNA template into high-quality mRNA, which is then rigorously validated for both integrity and protein expression using cell-based assays.

This streamlined workflow means that what once took months can now be accomplished in weeks. This rapid cycle reduces cost and resource expenditures while creating a dynamic environment where new data can swiftly be incorporated and acted on.

VelociVax is designed to integrate seamlessly with our broader oncology portfolio. In preclinical models, our mRNA constructs are being evaluated in combination with our bispecific antibodies and checkpoint inhibitors. The idea is simple yet powerful: by priming the immune system to target tumor-specific neoantigens and then release the brakes on immune cells with checkpoint blockade, our goal is to orchestrate a more robust antitumor response.

Looking Ahead

The implications of VelociVax extend beyond its immediate applications. While our initial focus has been on oncology, VelociVax’s utility is not limited to cancer alone. We can also use the technology to design mRNA vaccines with the potential to promote immune tolerance—training the immune system not to attack the body’s own cells—in hopes of helping prevent autoimmune diseases or other immune-related conditions like chronic hepatitis. This versatility is key to our long-term vision, as it positions Regeneron to respond quickly to emerging health challenges by generating custom-tailored mRNA solutions.

Though VelociVax is currently a research tool, its impact is already being felt across Regeneron’s research and preclinical pipelines. Our goal is to continuously refine this platform, using the insights gained from each study to drive further innovation. In time, we expect VelociVax to not only support but also accelerate the translation of groundbreaking mRNA-based immunotherapies from the lab bench to the clinic.

VelociVax represents more than just an mRNA generation tool—it is a critical enabler, one that empowers our teams to innovate faster and smarter as we work to move science to medicine.

All therapies discussed herein are investigational and have not been evaluated by any regulatory authority.