The Regeneron Scientific Journey: Mapping the Path from Human Data to Potential New Medicines

From early discovery to preclinical research, science and data power the journey to life-changing medicines.

Regeneron

June 09, 2026

Medicines don’t just start in the clinic. Their journey begins long before a clinical trial, with insights into human health and disease thoughtfully sourced from real people. Our science to medicine journey is guided by asking the right questions: with robust human datasets, deep biology expertise and cutting-edge technology, we turn discovery into potentially life-changing medicines for people with a wide range of diseases.

Target Discovery: What are the root causes of human health and disease?

To best understand the root causes of human health and disease, we start with people. Equipped with trusted, deidentified human data at scale— thoughtfully sourced from millions of volunteers across a wide range of populations—our genomic research powerhouse, Regeneron Genetics Center^® (RGC^®), collaborates with academic institutions, health care systems, nonprofit partners and other organizations across the globe to enrich our knowledge base and deepen biologic insights.

Multiomics provides a broader view of a patient population

Our lab technicians, data scientists and bioinformaticians collect and analyze large amounts of data using cloud technology to simultaneously look at genes, proteins, health records and more. RGC researchers use different kinds of biological data to find out how changes in our genes and proteins affect whether we get sick, stay healthy or recover. By analyzing so much data with advanced tools, we can discover important details about diseases that would be hard to find otherwise.

Many promising drug candidates in Regeneron’s pipeline began with RGC target identification or were informed by RGC insights in further research. Scientific discoveries from RGC have illuminated the genetic and molecular drivers of conditions spanning cardiovascular, metabolic, inflammatory, neurological and rare diseases, providing a powerful foundation for developing new potential medicines to serve patients in need.

Watch this video to learn how we deep dive into human genetics

Target Validation: What role does this potential target play in health or disease, and can it be addressed to benefit patients?

Once researchers identify a molecular clue linked to health or disease, the next challenge is to demonstrate that it truly makes a difference in disease biology. Regeneron’s diverse teams of scientists get to work validating potential targets: our library science team reviews scientific literature for existing research on potential targets to inform our validation strategies, while multiple teams of research scientists recreate certain biological conditions in the lab to learn more about them.

Regeneron research scientists use cell-based (in vitro) and animal (in vivo) models to understand how a potential target truly influences disease biology. Our proprietary VelociSuite^® technologies, like VelociGene^® and VelociMouse^®, are used to precisely modify genes in mice to replicate human disease, allowing scientists to observe how genetic drivers and molecular pathways behave in living systems. By inactivating genes or modifying specific targets in mouse models, we can see how those changes alter biological processes and reveal how diseases may develop in people.

This rigorous validation process ensures that only the most promising targets for treating patients advance in the journey towards a new potential medicine—a critical checkpoint bridging discovery and development.

Therapeutic Modality Selection: How should we go about treating the disease by focusing on this target?

Regeneron is uniquely equipped to tailor our therapeutic approaches to the needs of each disease and the people impacted, matching the specific therapeutic target to the technology or combination of technologies best suited for effective treatment:

Protein therapeutics (i.e., antibody therapies like monoclonal antibodies, bispecific antibodies, and altibodies)
Genetic medicines (i.e., gene editing, gene silencing, gene therapy)
Cell therapies (designed to harness the body’s own cells for treatment)

This versatility ensures that every medicine is designed around the science and the patients themselves. The depth and breadth of technologies and capabilities allow our scientists to think creatively and select the most effective approach(es) based on disease biology, mechanism of action and patient needs.

Preclinical Testing: How does this drug candidate work in treating disease?

In order to determine how a drug candidate may treat disease, and before any new medicine can reach a patient, it must first be thoroughly vetted in preclinical development. This is where scientific hypotheses become tangible drug candidates that must be tested in disease models. Other technologies in Regeneron’s VelociSuite^®, like VelocImmune^®, VelociMab^® and Veloci-Bi^®, accelerate this process, enabling rapid generation and screening of new therapeutic candidates.

Watch this video to see our scientific research in action

Scientists once again use in vitro and in vivo experiments in cell and mouse models of disease to explore how candidate medicines interact with their targets and influence disease progression. They also use ex vivo studies using cells taken from disease models or patient samples to ensure that the drug candidate functions as expected when it’s ready to be tested in humans.

Preclinical studies are critical for evaluating the mechanism of action, pharmacologic behavior and preliminary efficacy and safety of emerging drug candidates to determine which ones meet the standards for use in patients. The result is a shortlist of thoroughly tested, data-driven drug candidates that can proceed to clinical trials, all with high confidence and deep knowledge of how they may work in the intended patient population.

Multiple Journeys to the Same Destination

From decoding DNA to testing molecules in disease models, Regeneron’s scientific journey is a continuous cycle of discovery, validation and refinement. Each stage informs the next, creating a feedback loop that helps science move faster and more precisely toward potential treatments for patients.

Our journey is powered by data and technology, alongside human expertise and ingenuity. And while every journey is different, they all share the same goal: turning biological and genetic insights into medicines that can potentially change lives.