Target Safety Assessment

A thorough understanding of drug target biology and its relationship to disease and normal physiology is an important part of the drug discovery and development process. Most drug targets are proteins that can become dysregulated in disease settings such as cancer but also perform critical roles in healthy tissues. Their modulation can therefore lead to on-target adverse events, where engagement of the target results in unintended side effects. Such target-related toxicities remain a major cause of drug attrition and setbacks during clinical development. By assessing the potential downsides of target modulation early, researchers can anticipate risks, plan mitigation strategies, and make more informed decisions throughout the discovery process. This is where the Target Safety Assessment (TSA) comes in, providing a structured, evidence-based framework for evaluating potential risks before costly development begins.

A TSA is a structured evaluation of the potential safety risks associated with modulating a biological target. It integrates data from a variety of sources, including clinical trials, scientific literature and omics data, to anticipate the potential adverse effects of target modulation. Typically conducted early in discovery, TSAs may be updated iteratively at key stages throughout the R&D process to capture safety risks as they emerge. By identifying potential safety liabilities early and proposing mitigation strategies, TSAs help to guide drugs through the discovery and development pipeline. However, several scientific and analytical barriers can hinder the generation of consistent and comprehensive TSAs. A key challenge is the complexity of integrating heterogeneous data sources and the time-intensive manual curation required to derive insights across different reference molecules, genetic models, and mechanistic information, which can extend timelines, limit scalability, and delay critical safety decisions.

To overcome these obstacles to TSA production, Onesum offers a scalable, technology-enabled service that combines advanced AI capabilities with scientific expertise to meet the stringent demands of target safety assessment. Working with cross-industry partners, Onesum is building standardized workflows for deploying LLMs that ensure TSA outputs are accurate, reproducible, and transparent. Its proprietary AI platform powers this service, producing audit-ready TSA reports at scale. This hybrid model of technology and human expertise delivers cost-efficient, scientifically robust TSAs at scale, enabling early identification of target-related safety liabilities and supporting confident decision-making across discovery and development.