Research

From the Genome to Drugs

Drug Discovery Research

Developing Targeted Therapies
Bringing innovative drugs to market requires a true commitment to the science of drug discovery. We have established therapeutic area research teams in metabolism, immunology, and ophthalmology.  Research projects are organized across functional areas tapping experienced biologists, chemists, and drug developers to advance molecules to clinical development.  All of our drug discovery programs are focused around gene knockout-validated drug targets were identified in our Genome5000TM program. Since most drugs act as antagonists of their targets blocking protein activity, our mouse knockouts serve as genetic models of drug action that guide our drug discovery process. In this way, we are able to make predictions about what a drug could do, before we invent the drug.  Our advantage in drug discovery research derives from this conceptual framework.

Genome5000 Program
Initiated in 2001, our Genome5000 program was designed to discover the function of nearly 5,000 mammalian genes through comprehensive phenotyping of gene knockout mice.  We focused our efforts on genes from the human genome that encode “druggable” targets, that is, those genes encoding proteins with the greatest potential pharmaceutical applications as either small molecule or antibody drug targets. 

Using both gene trapping and gene targeting technologies, we have studied the functions of 4,650 genes as complete gene knockouts in mice. We have also engineered hundreds of more complex and subtle mutations including conditional alleles, point mutations, and humanized alleles. Having completed the Genome5000 program in 2007, we have identified more than 100 drug discovery targets in a wide range of therapeutic areas. 

After identifying the most promising drug targets, our research teams focus on discovering and developing therapeutics that can hit those targets, We use state-of-the-art chemistry technologies combined with medicinal chemistry expertise to identify drug candidates suitable for preclinical and clinical testing. Using this approach, our scientists and clinicians  have successfully advanced numerous drug candidates into human clinical trials.

From the Genome5000 program, our scientists have gained a thorough understanding of target biology and identified new points of intervention for future therapies, Our unique knowledge of genetics has allowed us to better evaluate and advance our drug candidates in clinical trials. We believe that our systematic, genetics-driven approach will enable us to substantially expand our clinical pipeline and achieve successful commercialization of important new medicines.