The high levels of investment in pharmaceutical R&D over the past decade have not correlated with the number of NMEs gaining approval. Whereas poor PK/PD properties used to be the primary cause of attrition, most NMEs vs novel targets now fail due to lack of efficacy and/or safety in patient studies. So how can we assure that the right target is selected at the beginning of a drug discovery campaign? And how can we ensure validation enables translation into the clinic? This session covers a range of methodologies and case studies describing key approaches to address these questions.
Sanj Kumar, ELRIG
The translation of emerging knowledge of cancer biology into novel therapies remains a great challenge in drug discovery, and requires early collaboration between key stakeholders in academia and industry to ensure that the most relevant and actionable targets are taken forward. This session will focus on academia-industry collaboration models established in recent years, with sharing of examples of successful outcomes and lessons learned. A secondary focus will be on epigenetic mechanisms in cancer where pioneering work from organizations such as the Structural Genomics Consortium, has revealed a large number of potentially druggable proteins involved in the writing, reading and erasure of epigenetic marks.
The renaissance in the use of phenotypic cell-based assays throughout the drug discovery process is an opportunity to combine an approach used intensively in the pre-genomic area with enabling new technologies. Success in the area aligns drug discovery hypotheses with translational disease biology, leading to an anticipated improvement in attrition rates of new medical entities in the clinic.
Cellular imaging is a broad field that is being utilised to deliver multiple phenotypic assay opportunities in drug discovery programs (including simple to complex cell cultures, data analysis, and biomarker/phenotype detection), and is advancing rapidly.
The Phenotypic discovery and cellular imaging session at ELRIG Drug Discovery 2014 will cover progress in imaging technologies, analytics and cell biology in this lively and quickly progressing field.
The need to create chemical leads for ever more challenging biological targets has led to a need for increased chemical innovation in lead discovery. Recent innovations include the generation of high quality hits outside classical ‘small molecule’ chemical space, the application of emerging synthetic methods and design principles to deliver diverse compound libraries, and greater consideration of conformational and kinetic profiles of potential hits. This session will highlight scientific approaches that increase chemical innovation in lead discovery which, together with novel partnership approaches, may deliver high quality leads for today’s challenging drug targets.
Cardiovascular and metabolic disorders are the predominant cause of death of disability in the industrialized world and increasingly prevalent in developing nations. Here, we address unmet needs and therapeutic opportunities including: a chemical biology approach to muscle cell lipotoxicity; human stem cell-derived cardiomyocytes as a platform for target validation in suppressing cardiac muscle cell death; genetics, genomics, and epigenomics of metabolism; signalling networks in glomerular kidney disease; nanotechnology and drug delivery; and chemically modified mRNA, a new technology for drug discovery and therapeutics.
In recent years, drug discovery has moved towards more complex assay technologies that address questions of direct relevance to human disease. These include: (i) the use human stem cell technologies for drug screening; (ii) the establishment of high throughput assays that allow drug discovery to be informed by ligand-binding kinetics; (iii) the evaluation of methodologies to exploit the clinical potential of ligand bias and (iv) platform technologies to rapidly scan disease pathways for novel drug targets. The Innovation in Assay Development & Screening session at ELRIG Drug Discovery 2015 will cover major developments in this fast moving field.