The promise of open innovation in drug discovery: an industry perspective.

Abstract

The last decade has seen a huge increase in the number of academic drug discovery centers with the expertise to discover lead molecules, often termed chemical probes, with the pharmacological properties to test a biological hypothesis in disease models [1]. This has required the creation of academic compound libraries and high-throughput screening (HTS) centers, alongside growth in expertise in techniques including assay development, fragment-based lead generation and computational and medicinal chemistry. The most significant examples of these have been the NIH-sponsored Molecular Libraries initiative, which created a number of HTS centers across universities in the USA [2], and the European Lead Factory (ELF) [3]. Alongside this, universities and charities have established drug discovery groups including Medical Research Council Technology and Cancer Research Technology in the UK, and the EU-OPENSCREEN project, which aims to establish a co-ordinated network of 15 or more screening centers across Europe [4,5]. The common objective of these groups is to source novel target hypotheses from academia, to generate lead molecules and to use these molecules to validate the target in cellular and animal models of disease, with the ultimate objective of validating new targets and creating a drug discovery program. While there have been notable examples of the delivery of candidate drugs in rare and orphan diseases relatively few academic drug discovery projects have progressed into the clinic. Reasons for this include the low levels of target validation associated with academic drug targets, the quality of the compound collections and hence lead molecules identified and the lack of expertise, and funding, within academia to deliver drug candidates and support clinical studies. These gaps in capability can be addressed through collaboration with pharma, while the gap in