ABSTRACT
Support is requested for a Keystone Symposia conference entitled Phenotypic Drug Discovery: Recent
Advances and Insights from Chemical and Systems Biology, organized by Drs. Mark Mercola, Fabien Vincent,
Monica Schenone and Arsenio Nueda. The conference will be held in Breckenridge, Colorado from March 3-7,
2019.
Unlike conventional target-centric drug discovery, Phenotypic Drug Discovery (PDD) places its focus on disease-
relevant phenotypes and agnosticism with regard to molecular mechanism of action. Thus, it offers unique
opportunities in terms of discovery of novel biology and first-in-class therapeutics but is matched with significant
challenges, a number of which can be addressed by recent advances in chemical and systems biology. A key
aspect of the conference will be to share information and processes on how best to employ phenotypic strategies
to discover novel biology and effectively prosecute drug discovery programs. Specific topics will include:
chemical biology advances and case studies in target identification; case studies of recently advanced clinical
candidates and approved drugs; functional genomics and systems biology advances and case studies; project
prosecution including lessons learned from lead optimization and pre-clinical development; complex cell-based
models and new assay modalities.
With few non-commercial meetings covering drug discovery as a discipline, this conference has the stature and
breadth to bring together accomplished and influential scientists from industry and academia as demonstrated
by the 2016 conference. Its scope will be broader than other PDD and Chemical Biology meetings since it will
cover the entire range of activities and technologies from phenotypic assay systems, to target identification and
FDA approval of novel therapeutics. Given that there is still an emphasis on target centric discovery in
pharmaceutical companies, this conference can be instrumental in further promoting a shift in mindset and
contributing to the consolidation of PDD as an integral part of the drug discovery paradigm. The vision for this
conference would be to share success stories of newly approved drugs and late stage clinical candidates, along
with key lessons and best practices, to inform future PDD projects.