Chemical biology is gaining increasing attention as a critical component of modern biomedical research. The increasing costs of drug discovery have led to a consolidation trend within the pharmaceutical industry and a clear shift of resources away from early discovery activities towards the later stages of the drug development process. In a way this is a paradox. The decoding of the human genome has spawned a wealth of information of new protein-encoding genes in the human body. However, it has become increasingly more apparent that further research into factors determining gene expression, epigenetic effects and functional proteomics are required to understand the complexities of biological mechanisms and causes for disease.  

In an attempt to unravel the complexities of disease-related protein function, there has in recent years been a steady rise in the number of reports of antibody-based inhibition of protein targets and gene-modified or RNAi-induced regulation of gene-expression. However, it has become obvious that gene knock-down and knock-out models, as well as antibody inhibition of target proteins, are not physiologically equivalent to pharmacological modulation of a target protein by a small-molecule. This realisation is at the heart of the rising demand of chemical biology research. The drug discovery pipelines of the pharmaceutical industry rely heavily upon knowledge generated in the academic sector, and this trend is expected to become even more critical in the future. Hence, the role of academic research is increasingly critical with respect to the understanding of fundamental biology, validation of drug targets, and as an essential part of the drug discovery value-chain.   

 

Small-molecules serve as an excellent complement to transgenic and RNAi tools in that they target the  protein gene product rather than the gene locus or mRNA, have virtually limitless structural diversity, can be made reversible, can affect target functions for defined periods in isolated proteins, cells, or organisms, and can serve as either inhibitors or activators of protein function. In addition, the sciences of medicinal and synthetic organic chemistry are mature disciplines and the techniques and concepts are well understood, allowing for efficient development of chemical probes for research purposes.

Unfortunately, most Swedish academic institutions have only limited access to the tools required to develop high-quality small-molecules, primarily due to the prohibitive cost associated with the activities.  Although there are a number of commercial ventures and organisations that are fully capable of providing this expertise to academic groups, the costs of these services are significant and usually restricted to the pharmaceutical industry or endevours strictly focused on the development of therapeutics.  Thus, the possibilities for identification and use of small molecules in purely academic study of fundamental biological processes is limited.  CBCS aims to address this need.

In collaboration with CBCS, academic research groups have the opportunity to utilise state-of-the-art technology platforms (assay development, screening of small-molecule libraries, chemical optimisation, etc), traditionally only available to the pharmaceutical industry. Our organisation can thus provide access to small molecules enabling exploration, and facilitate better understanding of both fundamental biology and disease-related processes. Consequently, these efforts will promote new therapeutics to be developed more efficiently within the pharmaceutical sector.