Development of novel chemical drugs is an incredibly complex and time-consuming process, including the validation of therapeutic targets, the establishment of biological models and methodologies for in vitro and in vivo screening of lead compounds, pharmacokinetic and pharmacodynamic studies, preclinical safety evaluation in animal models and the potential for differences in the human body, and multiple phases of clinical trials. All this makes drug discovery and development a highly costly and risky business, with extraordinarily high failure rates. Most lead compounds are eliminated at later stages of research and development due to unsatisfactory drug efficacy or excessive toxicity, or a failure to display obvious advantages and improvements over existing drugs. It is vital to build a comprehensive, rapid and effective evaluation system in the early stages of R&D that can accurately predict the potential clinical efficacy and drug safety of lead compounds, enabling scientists and managers of new drug development to make decisions scientifically as soon as possible.

The selection of lead compounds that minimize risk at pre-clinical and clinical trial stages is a major challenge in the field of international novel drug research and development. Chipscreen Biosciences was the first in the world to establish an integrated drug discovery and early evaluation system based on chemical genomics. The potential molecular pharmacology and toxicology of new candidate compounds is evaluated and predicted through a parallel chemical genomics analysis of the expression data of genes associated with known compounds (successful or failed drugs) sharing structural features with the candidate. Promising candidates undergo continuous structural optimization, ensuring that only those lead compounds with the best overall evaluation performance enter the next stage of drug development, which is crucial for reducing risk. This process reflects the Critical Path Initiative advocated by the FDA to promote the success rate of innovative drug development (as shown in the Figure).

Our genomics-based screening platform underpins the core competitiveness of Chipscreen Biosciences, and provides strong technical support for sustained independent innovation in drug research and development here in China.