Our Research Platform

At our core, Agios is a science-driven research organization. We have built a discovery platform upon our expertise in the fields of cellular metabolism and precision medicine across three major focus areas: cancer metabolism, rare genetic diseases and metabolic immuno-oncology. We take a systems biology approach to deeply understand disease states, drive the discovery and validation of novel therapeutic targets, and define patient selection strategies, thereby increasing the probability that our experimental medicines will have the desired therapeutic effect.

Systems biology involves integration across different data types to achieve a better, more predictive understanding of complex and dynamic biological systems. By combining large-scale expression profiling, genomic and proteomic data sets together with our proprietary metabolism data, we can derive deep knowledge of the disease states and therapeutic targets of interest. Our proprietary metabolomics technology enables a more rapid and comprehensive study of metabolic pathways than could be accomplished in the past. Using this toolkit, we can interrogate both the steady state levels of metabolites in cells (akin to the number of cars at a specific point on the highway) and the rate of flow of metabolites through a cellular pathway (akin to the speed of cars going past a specific point on the highway). Put together with the other dimensions of systems biology, our metabolism platform provides an unprecedented level of understanding of therapeutic targets for complex diseases.

Deep Understanding of Metabolic Pathways Drives Development of Transformational Medicines

Cellular Metabolism

Cellular metabolism, which refers to the set of life-sustaining biochemical transformations of living organisms, is essential for cells to carry out their normal functions, and importantly, is substantially altered in a number of pathologic settings. These biochemical transformations are used to break down nutrients to create the energy that powers cells and to manufacture all of the building blocks that provide the structure and function of cells. The differences in metabolism in disease versus normal physiology create important therapeutic opportunities. We are leaders in discovering and validating potential new medicines to inhibit, restore or alter a cell’s metabolic state to treat cancer and rare genetic diseases.

Precision Medicine

Precision medicine involves the tailoring of medical treatment to the specific characteristics of an individual’s genetic content and the cellular and molecular profile of their disease. This approach allows the use of predictive biomarkers to select patients who are most likely to respond to a particular therapy, thereby dramatically increasing the probability of success in clinical trials. Our understanding of cellular metabolism within diseased tissues enables the development of methods to measure the effect of a drug on the target of interest and the patient, or pharmacodynamic markers, and patient selection strategies for clinical development. Utilizing our platform, we identify altered metabolic pathways within abnormal cells. Metabolism-based biomarkers can identify appropriate patients for clinical trials, serve as pharmacodynamics markers to characterize medicine/target engagement in patients, and permit the monitoring of patient response to therapy.

The Metabolome

Untapped Opportunity for Novel Precision Medicine