Approved Research

Integrative Genomics for Therapeutic Design and Prioritization

Lay summary

The creation of novel medicines requires two components: a biological "target" to be affected, usually a protein, and a technology that can alter it.  For nearly the entire history of pharmaceuticals, we were limited by technology.  We could only rarely create novel molecules that would bind to proteins or other biological actors and modify their function.  There were many more potential targets uncovered by researchers than could be effectively drugged.

Over the past 20 years, this paradigm has flipped.  We have a wealth of new modalities: antibodies, cell and gene therapies, synthetic RNA and beyond.  We know much less about where and how to apply these new technologies. 

Part of the answer lies in genomics. By linking DNA sequences with medical records and biochemical measurements, we can infer the relationship between genes and disease, giving evidence about how to target our new technologies.  Running these analyses is often complex and requires specialized computational training, and data access. We propose to build best-in-class genomic data tools uniting evidence across biological databases and sources to assist the deployment of novel biotechnologies and the development of the next generation of medicines.