People around the world who suffer from diseases for which there is inadequate treatment pin their hopes on modern science to find new therapies. As part of the Novartis Institutes for BioMedical Research our mission is to discover innovative medicines that address unmet medical need including diseases such as cancer, metabolic disorders, cardiovascular disease, inflammation and autoimmune diseases, CNS disorders, and infectious diseases.
Our oncology research group pursues two approaches to selectively kill tumor cells: 1) small molecule inhibitors of oncogenic drivers in genetically-defined patient populations, and 2) antibody-drug conjugates to selectively deliver cytotoxic payloads to cancers expressing the targeted tumor antigens on their surfaces. In a second, distinct strategy, we also pursue cancer immunotherapies designed to re-activate the body’s immune system to combat the tumor.
Technology plays a key role in executing our strategy. We exploit sophisticated screening systems to carry out complex cell-based assays against our collection of several million low molecular weight compounds. We also tap into in-house technologies developed for antibody discovery as well as our proprietary antibody-drug conjugate technology platform.
Cardiovascular diseases are the leading cause of death globally. Coronary heart disease, stroke and peripheral arterial disease account for the majority of deaths where the underlying mechanism involves atherosclerosis and hypertension. Identifying and targeting these conditions and other relevant disease processes underlying cardiovascular disorders such as dyslipidemia, atherosclerosis and vascular diseases, Type 2 diabetes, heart failure, and cardiac arrhythmias sit at the cornerstone of research within the GNF cardiovascular program.
Our researchers are creating new research methods to translate information about atherosclerosis and other complex cardiovascular diseases into clinically-relevant therapeutic strategies. By interrogating mouse and human cardiovascular phenotypes and genomic datasets our goal is to discover novel targets and create disease-modifying therapies that reverse the pathophysiology of cardiovascular disorders.
Today, metabolic disorders like obesity, diabetes and liver disease create a substantial health burden on society. So that we can discover new therapies to treat these diseases, our Metabolic Disease program is focused on a gaining a thorough understanding their pathophysiology. We have a particular focus on restoring beta cell mass and function, increasing glucose disposal in skeletal muscle and protection from liver damage associated with fatty liver disease.
Our infectious disease program is focused on some of the world’s most deadly, yet under-researched pathogens, including the related parasites that cause Chagas disease and visceral leishmaniasis. We also target new medications for viral respiratory diseases and other infectious diseases where there is a strong unmet medical need.
Tissues within the musculoskeletal system--muscle, bone, tendon, cartilage, and ligaments—are essential for motion as well as many other functions, including regulation of blood sugar and energy use. Damage from normal aging or as a result of traumatic injuries or other diseases leaves these tissues with less intrinsic ability to repair on their own leading to pain, impacted mobility and affected metabolic function.
Within the GNF drug discovery group, we are working to improve the repair of muscle, tendon and cartilage through the stimulation of the endogenous progenitors or stem cells residing in each of these tissues. Our goal is to develop therapeutics that will accelerate and improve the natural course of healing and reconstruction of damaged tissue to ultimately improve mobility, reduce pain, and increase the quality of life for patients.
Hematopoietic stem cell (HSC) transplantation is a promising treatment option for both malignant and nonmalignant diseases. The Regenerative Medicine group identifies novel regulators of HSC fate and is exploring new ways of improving hematopoietic stem cell growth and engraftment.
Scientists are also investigating new gene editing approaches to develop new therapeutic strategies for genetic disease. Although still in its infancy, genome editing and correction present new opportunities for tackling a number of diseases that are beyond the reach of previous therapies.
Despite tremendous progress in recent years, there remains high unmet medical need for people suffering from common respiratory diseases, like chronic obstructive pulmonary disease (COPD), asthma, and rarer disorders such as cystic fibrosis.
We are using a diverse range of discovery tools aimed at identifying biologics or small molecules for the treatment of lung disorders. Our novel approaches focus on mucus control, reducing abnormal immune responses, and improving cell regeneration to discover safe and efficacious medicines to improve the lives of patients with respiratory diseases.