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Drug Discovery
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.
Accordion: Cancer
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 Disease
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. Metabolic Diseases
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. Infectious Diseases
Our infectious disease program is focused on some of the world’s most deadly, yet underresearched 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.
Musculoskeletal Disorders
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. Regenerative Medicine
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. Respiratory Disease
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. Source URL: https://www.gnf.nibr.com/our-research/drug-discovery