Early stage drug discovery: Target identification and validation

Unlock the potential of your drug discovery journey with our comprehensive target identification and validation solutions. From disease relevance to druggability, we're here to set your project on the path to success.

Lay the right foundation for your 
drug discovery

Drug discovery starts with identifying and validating the right target. We understand that this critical first step lays the foundation for the entire drug discovery process. Our team of expert scientists employs a rigorous and multi-faceted approach to target identification and validation, ensuring that your drug discovery efforts are built on a solid scientific foundation, de-risking your development path and increasing the likelihood of success.

By thoroughly investigating several key attributes, researchers can build a strong case for pursuing a particular drug target, increasing the likelihood of developing effective and safe treatments. Our target identification and validation workflows assess these critical factors, which include:

Understanding target biology:

Drug targets are critical components of cellular signaling pathways that lead to disease. Thorough studies should be conducted to understand how these targets function under both normal and disease conditions. A strong correlation between changes in target activity and disease severity builds a compelling case for the target’s role in the disease process.

Gene and protein expression analysis

Examining the spatial and temporal expression of targets in relevant tissues or cells provides valuable insights into their role. Comparing gene expression between healthy and diseased samples can highlight the target’s therapeutic potential.

Cellular signaling pathway analysis

Cell signaling pathways involve complex networks of proteins. Phenotypic and functional assays can reveal a target’s position and function within these pathways. Studying these pathways with and without target modulators helps assess therapeutic potential and possible side effects.

Genetic evidence

Animal models, typically mice or rats, with gene knockouts (KO), can provide evidence linking the target to the disease. Additionally, human subjects with relevant genetic mutations can offer insights into the target’s role, suggesting the gene either contributes to the disease or its absence may be beneficial.

Knockdown studies

Using tools like small interfering RNA (siRNA) or short hairpin RNA (shRNA) to reduce gene and protein expression can directly demonstrate the relationship between the target and observed phenotypic effects, establishing cause-and-effect connections.

Pharmacological evidence and preclinical studies

Testing target modulators (e.g., small molecules, degraders, antibodies) in cellular and animal models that mimic human disease helps predict whether targeting the molecule leads to a desired therapeutic outcome and also helps assess the impact of target perturbations.

Druggability assessment

Early in the target validation process, it’s crucial to determine whether the target can be effectively modulated. The modulation can be with small molecules, antibodies, or specific degraders.

Clinical and human proof of concept

Real-world clinical data from subjects treated with target modulators provides invaluable evidence of the target’s potential. This data also helps assess side effects and design strategies to mitigate them.

Off-target effects evaluation

Precision medicine aims to selectively affect desired targets while minimizing adverse effects. Evaluating off-target effects is essential for anticipating and preventing potential safety issues.

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Fueled by our continuous innovation and armed with EnzeneX™, our fully-integrated CDMO solutions and our biosimilars pipeline are designed to help bring your biologics innovations to life.

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