We invent and research potential new medicines for a broad range of serious conditions, including retinal eye diseases, cancer, rheumatoid arthritis, asthma, atopic dermatitis, pain and infectious diseases. Our industry-leading antibody technologies and scientific teams enable us to identify and study dozens of potential drug candidates simultaneously and with great efficiency. The best of these candidates then progress into thorough preclinical and clinical research phases, building our completely homegrown portfolio of over 30 investigational medicines.
Putting patients first
At every stage of research, from early genetic research through bringing potential medicines through clinical development, we work closely with patient advocacy groups and the specific disease communities to listen, understand and apply their experiences to our work. These insights help us learn from those most intimately impacted by certain diseases, ensuring that we help meet their needs. This guidance especially helps to inform our clinical trial designs, clinical trial recruitment processes and therapeutic delivery systems. We celebrate and appreciate our relationships with each unique disease community and are proud to support the efforts of advocacy organizations.
Cardiovascular and metabolic disorders encompass a large number of diseases, including coronary artery disease and diabetes. Our clinical programs focus on treating cardiovascular disease, the number one cause of morbidity and mortality in the United States and other developed countries, and on muscle-growth disorders.
We approach early-stage research for cardiovascular and metabolic disease interventions in multiple ways:
- The complement cascade is a component of the immune system effective at clearing pathogens from the body. When dysregulated, however, complement can damage cells and organs. We are developing
complement inhibitorsfor diseases like paroxysmal nocturnal hemoglobinuria.
- We are also researching both forms of hypercholesterolemia through different mechanisms, including using fully-human monoclonal antibodies to target proteins that contribute to lipoprotein metabolism.
- Using agonist antibodies that target the leptin receptor, we aim to help correct the metabolic consequences of abnormalities in leptin signaling.
- More than two decades of our research has helped us understand the biology and natural history of the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), an ultra-rare and debilitating genetic disease that starting at a young age, progressively replaces muscle, tendons, ligaments and fascia with bone (heterotopic ossification or HO). We are investigating a fully-human monoclonal antibody that binds to and neutralizes Activin A, which we have discovered plays a key role in FOP by driving HO.
We use our VelociSuite® technologies to rapidly discover and develop new antibody combinations to address, treat or prevent serious infectious diseases.
In an increasingly interconnected world, infectious diseases are able to spread more quickly than in the past, enhancing their threat to global health and elevating the need for new treatments that are safe, effective and more resistant to new mutations.
We leverage our proprietary VelociSuite® ‘rapid response’ technologies to create novel antibody cocktail fully-human monoclonal antibody treatments, with the goal of addressing a number of common and newly emerging infectious diseases, such as COVID-19, Ebola and the Middle East respiratory syndrome (MERS) coronavirus.
The immune system plays a critical role in many facets of human health: a vast range of cells, diverse proteins, lipids and signaling pathways that have evolved to control infections, respond to injury and help the body recognize self from non-self. The immune system is naturally and finely poised to rapidly respond to ensure that bacteria, viruses, parasites and even pre-cancerous cells, are quickly identified, targeted and cleared. Issues arise, however, when the system becomes dysregulated, either promoting an uncontrolled ‘over’ response or, inversely, failing to respond and protect the body appropriately.
Our research focuses on understanding the cellular and molecular mechanisms of the immune system that relate and drive the pathology of diseases such as asthma, inflammatory bowel disease, autoimmune diseases like lupus and type 1 diabetes, and cancer. This helps us to identify therapeutic options that address unmet medical needs in many such diseases. For example:
- In cancer immunology, we seek to understand and target immune checkpoints and leverage the power of bispecific antibodies to utilize the immune system to clear tumors and promote long-term survival of patients.
- We are investigating new molecular pathways involved in skin and gut inflammation and epithelial repair for the treatment of diseases such as pustular psoriasis, ulcerative colitis and Crohn’s disease.
- We continue to explore new pathways and mechanisms that promote ‘type 2 immunity’ in severe allergy, asthma and atopic dermatitis and to understand the impact of this pathway on food allergies and celiac disease.
- We develop new models of inflammation to explore mechanisms behind immune tolerance, such as graft versus host disease and organ transplant rejection. Using tools such as Veloci-T™ mice, we are also studying immunity related to T cells and other groups of genes.
Using the above research approaches, we have developed FDA-approved medicines for the treatment of diseases such as atopic dermatitis, asthma, rheumatoid arthritis and advanced cutaneous squamous cell carcinoma. Given the interconnected nature of these conditions, we have an ever-deepening clinical portfolio exploring new options for the treatment of allergy, autoimmune diseases and even cancer immunology.
Despite decades of progress, cancer still remains one of the world's most serious health problems. We believe we can help cancer patients most in need of therapy with the benefits of immunotherapy.
PD-1 inhibition is the backbone of our oncology portfolio and is a widely recognized way to harness the body’s own immune system to fight and kill cancer cells. It has the potential to be combined with multiple innovative therapies for precisely tailored treatments. We are researching PD-1 inhibition as a monotherapy for various malignancies and in combination treatments with oncolytic viruses, vaccines and gene therapies, and other novel modalities.
Bispecific antibodies are another promising way to target cancer cells. We are advancing multiple investigational candidates created with our Veloci-Bi® platform, which allows us to create bispecific antibodies with no linkers or artificial sequences. Designed to closely resemble natural human antibodies and with similar phamacokinetics, bispecifics are designed to bind to two different targets, opening up a diverse array of possibilities for targeting and killing cancer.
- Our bispecifics fall into three main categories: CD3 bispecifics that help the immune system recognize cancer cells; CD28 costimulatory bispecifics that boost T-cell activation against cancer; and tumor-specific bispecifics that bind to multiple targets on the cancer cell surface to hamper their survival and proliferation.
Our clinical portfolio currently includes antibody candidates for over 30 types of cancer, including cutaneous squamous cell carcinoma, basal cell carcinoma, non-small cell lung cancer, cervical, diffuse large B-cell lymphoma, follicular lymphoma, multiple myeloma, ovarian, prostate cancers and many more.
Learn more about our costimulatory bispecific antibody research efforts in the Regeneron Perspectives blog
We are applying novel approaches to address hematological diseases with high unmet need–from aberrant clotting disorders, such as thrombotic events and hemophilia, to hematological malignancies such as lymphoma and multiple myeloma.
- In the area of hematological malignancies, we continue to expand the capabilities of our bispecific and costimulatory antibody platforms to transform the treatment of B cell malignancies. In the clinic, our CD20xCD3 and BCMAxCD3 bispecific antibody programs are in trials for the treatment of patients with B-cell non-Hodgkin lymphoma and multiple myeloma, respectively. These therapies have been designed to bind both the tumor cells and the patients’ T cells to trigger cell killing. Our scientists continue to create and explore novel combination therapies to further improve the durability of antitumor responses in these hard to treat malignancies.
- In addition to our work in hematologic malignancies, we are also growing our benign hematology pipeline to include potential medicines for a broad spectrum of hematological disorders. Our areas of research and clinical activity include clotting and thromboembolic disorders, anemias, hematopoietic stem cell transplantation (HSCT) and many other rare blood diseases.
- As an example, we continue to investigate the potential of our antibody, which blocks complement factor C5 to prevent the destruction of red blood cells (hemolysis) in complement-mediated diseases. We are exploring a first-in-class therapeutic directed at interleukin receptor-2 gamma (IL-2Rγ) for a potentially more effective means of immune suppression for use in multiple diseases (e.g., aplastic anemia). We have also recently expanded a collaboration involving CRISPR-Cas9 technology and our adeno-associated viral vector engineering capabilities to potentially treat hemophilia A and B.
Our Anti-vascular endothelial growth factor (VEGF) medicine is the global standard of care for certain serious retinal vascular diseases, and we are committed to continuing to advance the treatment of serious, vision-threatening diseases. We are developing and improving anti-VEGF therapeutics beyond the currently approved indications, as well as pursuing novel therapies for many other serious ophthalmic diseases, including glaucoma, uveitis, corneal dystrophies, dry eye and inherited retinal disease.
In addition to the proprietary Trap technology that was the basis for our leading anti-VEGF molecule, we use many cutting-edge molecular pharmacologic modes such as monoclonal antibodies, RNA interference and gene therapy, as the basis for new agents.
Learn more about how we are working to ‘see a new way’ for eye diseases.
Many people experiencing chronic pain are currently treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and opioid medicines, which may result in inadequate pain relief, intolerance and carry long-term safety risks. We are advancing novel treatment options for patients suffering from chronic pain conditions, such as osteoarthritis pain.
One such category is a novel class of proteins, called neurotrophins, which promote the growth of nerve cells. Members of this family of proteins include nerve growth factor (NGF). As a company founded by a neurologist, Regeneron has been involved in this area of study from its early days, with our first investigational drug – a neurotrophic factor – entering clinical development in 1992.
Learn more about the challenges of treating pain from one of our research scientists.
At Regeneron, we pursue foundational science that can impact diseases with both large and small groups of patients. To do so, we leverage cutting-edge research tools and unique resources, such as the Regeneron Genetics Center®, to further understand the genetic variations that may protect someone from a disease or make them more susceptible. We then use Regeneron-invented technologies to further understand the root causes of disease and identify potential new therapies. Through this approach, we have created numerous approved and investigational medicines for people with rare and ultra-rare conditions.
Our very first FDA-approved drug, launched in 2008, treats aspects of Cryopyrin-associated periodic syndromes (CAPS), a group of rare illnesses characterized by lifelong, recurrent symptoms of rash, fever/chills, joint pain, eye redness/pain and fatigue.
Areas of ongoing research for rare diseases include:
- Fibrodysplasia ossificans progressiva (FOP), a progressive, severely disabling, life-altering disease in which muscles, ligaments, tendons and other connective tissues are transformed into bone. Scientists in our Skeletal Diseases Therapeutic Focus Area have been investigating FOP and related conditions for more than 20 years. Based on our novel discoveries about the functioning of this ultra-rare disease (with only 800 known patients in the world), late-stage clinical trials of a Regeneron-invented investigational treatment are currently underway.
- Homozygous familial hypercholesterolemia (HoFH), an inherited form of extremely high cholesterol causing inability to process the body's natural supply of cholesterol in the liver. Very high levels of LDL cholesterol can block arteries (atherosclerosis) and lead to a heart attack or stroke at a very young age. We are exploring various approaches in HoFH, including an investigational, fully-human monoclonal antibody that specifically binds to and blocks a key protein in regulating LDL cholesterol levels.
- Paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, chronic, life-threatening disease in which genetic variants lead to increased risk for destruction of red blood cells, resulting in a range of symptoms including fatigue, shortness of breath and blood clots. We are exploring an investigational, fully-human monoclonal antibody designed to block and prevent the destruction of red blood cells that cause the symptoms of PNH and other diseases mediated by abnormal complement pathway activity.
- Lipodystrophy, a rare metabolic disorder characterized by decreases in the quantity and distribution of body fat, which is often associated with low levels of a hormone called leptin. Low leptin leads to extreme hunger, disrupts the body’s metabolism and can cause fatty tissue to accumulate in muscles and organs such as the liver. Lipodystrophy can be inherited or acquired and affect people of all ages. We are testing a novel antibody that stimulates the leptin receptor to replace the deficient hormone. We hope to learn whether this therapy improves the health of people living with different types of lipodystrophy.
Learn more about how we listen and learn from the rare disease patient community.