Core Components of a Small Molecule Target Service A comprehensive service follows the early drug discovery workflow: 1. Target Identification & Prioritization Bioinformatics & Omics Analysis: Mining genomic, proteomic, and clinical data to find proteins or pathways dysregulated in a disease. Genetic Screens: Using CRISPR-Cas9 or RNAi to knock out/knock down genes and identify which are essential for disease cell survival. Literature & Database Mining: Systematic review of existing scientific and patent data to propose novel or repurposable targets. 2. Target Validation In Vitro Models: Confirming the target's role in disease using engineered cell lines (overexpression, knockout) and relevant disease models (e.g., cancer cell lines, neuronal cultures). In Vivo Models: Using animal models (e.g., zebrafish, mice) to see if modulating the target (genetically or with a tool compound) has the desired therapeutic effect and is safe. Biochemical Validation: Demonstrating the target protein is expressed, has the expected activity, and is "druggable" (has a pocket where a small molecule can bind). 3. Assay Development & Screening This is a critical service. Providers develop robust tests ("assays") to measure target activity. Types of Assays: Biochemical Assays: Test compound binding/interaction with the purified target protein (e.g., enzymatic activity, protein-protein interaction). Cell-Based Assays: Test compound function in a living cell (e.g., reporter…
