Abstract

The future treatment for many of today’s diseases lies in discovering new biology, understanding and challenging scientific hypotheses, to develop novel therapies. Improving R&D productivity is critical to the success of drug discovery, with failure rates from pre-clinical development to launch often in excess of 90 percent. The advent of data science and artificial intelligence, coupled with ever more sophisticated platform technologies, such as dynamic multi-omics brings with it an expanding list of pathways and targets to pursue - and with-it new drug discovery challenges.
Exploring novel drug modalities which complement existing strengths in small molecule and biologic platforms has the potential to open new therapeutic options, and to help us prosecute drug targets traditionally viewed as intractable. Over the past few years we have made significant progress across seven additional drug discovery platforms, complementing existing capabilities in chemistry and protein engineering. These include modified mRNA therapies, antisense oligonucleotides (ASOs), oligonucleotide conjugates, bicyclic peptides, proteolysis targeting chimeras (PROTACs), anticalins and CRISPR. By combining new technologies, focusing on medical understanding, challenging scientific hypotheses and driving strategic private and academic partnerships, we are making real progress in our ability to define and prosecute novel treatment approaches for a variety of difficult to treat diseases.