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Milestone 7.A

Expand existing and develop new systems biology and systems pharmacology research programs to build multiscale models of disease that will lead to the identification of networks/sub-networks as drug targets and readouts of therapeutic activity and advance the validation of existing and novel targets, rational drug repositioning and rational development of combination therapy.

Success Criteria

Launch at least 6 cross-disciplinary research programs that bring together experts in translational bioinformatics, computational biology, genetics, epidemiology, drug discovery and clinical research to develop a predictive model of the disease. Efforts should support:

  • development of computational tools and infrastructure to allow basic and clinical  researchers to query model in silico and validate it by using it for  patient stratification, predictions of efficacy, on- and off-target adverse effects.
  • identification of quantitative methods to access synergy between (1) multiple therapeutic agents and (2) pharmacologic/non-pharmacologic perturbations.
  • development of phenotypic screens (in cell based and/or animal models) to advance  rational drug repositioning and data-driven development of combination therapy based  on the ability of individual or  combinations of therapeutic agents to shift the network state away from disease.

Research Implementation Area
Drug Repurposing and Combination Therapy Development

Implementation Activities

Funding Initiatives

Research Programs and Resources

Relevant Recommendations