We are a leader in the pharmaceutical industry in terms of research and development, including the level of our investment. The discovery and development of a new drug is a lengthy process, usually requiring approximately 10 to 15 years from initial research to market introduction, including approximately six to eight years from Phase I clinical trials to product launch. At each of these steps, there is a substantial risk that a compound will not meet the requirements to progress further, which may trigger the abandonment of the compound after substantial investments.
Our Research and Development teams work together to deliver new and better medicines to patients in the shortest possible time.
Novartis Institutes for BioMedical Research (NIBR)
The Novartis Institutes for BioMedical Research (NIBR) is the global pharmaceutical research organization of Novartis. With an ongoing focus on diseases for which medical needs remain unmet, scientists and physicians at NIBR are dedicated to ensuring that Novartis maintains its strong pipeline.
NIBR has sites in Basel, Switzerland; Shanghai, China; Cambridge, Massachusetts; and three other US locations.
Discovering new ways to improve and extend people’s lives
We currently have major research efforts focused on addressing some of the biggest health challenges the world is facing.
Cancer Breast, lung, colorectal, kidney, pancreatic, bladder, melanoma, lymphoma and several types of leukemia
Diseases associated with aging Tendon and muscle weakness
Neurological disorders Autism, schizophrenia, bipolar disorder, and Alzheimer’s disease
Auto-immune disorders Psoriasis, Sjoegrens syndrome, and systemic lupus erythematosus
Ophthalmology Diabetic macular edema, retinitis pigmentosa, dry eye disease, age related macular degeneration, and glaucoma
Cardiovascular and metabolic diseases Dyslipidemia, atherosclerosis and vascular diseases, type 2 diabetes, heart failure, cardiac arrhythmias and associated disorders
Diseases of the developing world Malaria, chagas, human African trypanosomiasis and leshminiasis
Pioneering innovative technologies and approaches for discovery
To accelerate the discovery of new medicines we are pioneering efforts to use innovative technologies and approaches to drug discovery. Some examples:
Chemical biology Researchers have used the standard tools of biology and chemistry to develop many successful treatments, and we continue to employ them. But we recognize that these tools leave many drug targets – key proteins and nucleic acids known to play a role in disease – out of reach. We would like to hit these targets to fight disease, but they’ve dodged the conventional molecules in our arsenal. To address this challenge, we are blazing a new path: organizing our early discovery efforts around chemical biology. Chemical biology brings together experts from different fields – including biology, chemistry and computer science – to create new types of molecules and use them to probe biological systems. We have joined forces with researchers from the University of California, Berkeley, to form the Novartis-Berkeley Center for Proteomics and Chemistry Technologies, based in existing labs at Berkeley. The focus of this collaboration is on the development of new technologies for the discovery of next-generation therapeutics, pursuing the vast number of disease targets that have eluded traditional small-molecule compounds and are considered “undruggable.”
Genome editing Genome editing, which enables scientists to change the DNA inside cells, has become a powerful research tool and technology for engineering cell and gene therapies. Through collaborations with leaders in this field, our scientists are working to harness this transformative technology to develop new therapies.
CRISPR, an acronym that stands for clustered regularly interspaced short palindromic repeats, is an approach that allows scientists to precisely edit the genes of targeted cells. Think of CRISPR as a pair of molecular scissors capable of snipping DNA. The tiny shears are combined with an RNA-based targeting molecule that scans the genome for specific sequences and makes a controlled cut at a single site. In a short period of time it has proven to be a powerful tool for creating very specific models of disease for use in drug discovery and has great potential for use as a therapeutic modality for treating disease at the genetic level by deleting, repairing or replacing the genes that cause disease. We currently have a collaboration with Intellia Therapeutics that is using CRISPR technology in conjunction with hematopoietic stem cells to explore treatments for sickle cell disease and thalassemia.
Homologous recombination is a biological mechanism used by cells to ensure highly precise DNA repair. Our scientists are exploring the use of AAVs – adeno-associated viruses – that can be engineered to carry a specific genetic sequence and programmed to home to a target site in the genome. When they arrive, AAVs trigger homologous recombination, which overwrites a particular portion of a gene or even replaces an entire gene. We are currently exploring the use of this technology to develop treatments or diseases of the eye and blood.
Our work with both technologies is in a preclinical stage.
Immuno-oncology Tumors use molecular tricks to evade detection by the body’s natural defenses in our immune system. Our immuno-oncology research team is exploring new ways to harness this defense system to recognize and destroy tumors. The team is investigating three key steps in the immune response to cancer – education, activation and dissemination of immune cells to destroy tumors. They are seeking ways to improve immune cell activity at each step, and to combine these therapies with other treatments to offer the best outcomes for each patient.
Digital Digital technologies have incredible potential to unlock the next chapter of explosive medical innovation. We are applying digital technologies such as machine learning in drug discovery and wearable sensors in early clinical development to better understand patient outcomes, capture digital endpoints, and to test how patients respond to our therapies.
The exploratory phase of drug discovery
All drug discovery efforts at Novartis focus on patients. Scientists determine which diseases will be the focus of research efforts based on two questions: do we have, or can we gain, significant understanding of the cause, or mechanism, underlying the disease? And does this disease represent a significant unmet medical need? If the answer to both questions is yes, then Novartis develops a research program aimed at better understanding the disease and finding an effective therapy. Early-discovery science determines disease cause at the molecular level, using our own discoveries as well as those from external collaborators. We look for clues in both patients’ experience of the disease and the compendium of historical medical and scientific knowledge, integrated with the growing knowledge of human biology, chemistry and genetics.
Target discovery and drug design
Typically, making a drug begins with identifying a protein associated with human disease. These proteins are known as “targets”. When it is confirmed that a target plays a role in a disease, an experiment known as a high-throughput screen is conducted to find a chemical compound or antibody that binds to or “hits” the target in a way that alters the disease. Once chemical compounds or antibodies are identified by their binding to a target, they are enhanced to improve their safety and effectiveness. The resulting chemical compound or antibody becomes a drug candidate.
Preclinical safety and efficacy
An initial profile of a drug candidate’s safety and effectiveness must be determined before it is tested in humans. In this phase, scientists use computer models and laboratory tests to assess the safety of a drug candidate. These tests determine how well a drug candidate is absorbed, where it goes within the body, how it is broken down or metabolized, and how quickly and in what manner it is eliminated from the system.
Proof of Concept
All drug candidates are taken to the clinic via “proof-of-concept” trials to enable rapid testing of the safety and efficacy of the drug while collecting basic information on pharmacokinetics and tolerability, and adhering to the guidance for early clinical testing set forth by health authorities. Following proof-of-concept, our Global Drug Development organization conducts confirmatory trials on the drug candidates.
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