Target Validation
Target validation ensures that the target is expressed and then confirms if the engagement of the target has any potential therapeutic benefit. It is a critical step in the early phase of drug development.
Our scientists at Cellomatics offer target validation assay design and support for a wide range of projects.Â
Target validation includes the following stages:
- Discovering a biomolecule of interest
- Investigating the expression of the molecular target within the tissue of interest
- Evaluating its potential as a target to understand if it is directly involved in the disease process
- Designing a bioassay to measure biological activity to verify if target modulation produces the desired therapeutic effect
- Constructing a high-throughput screen
- Performing screening to find hits and evaluating the hits
- We can offer support and assay design for protein and novel target expression in a range of normal and diseased tissue, including:
- IHC and novel marker assay development & co-localisation studies
- Phosphorylated marker assay development
- High-throughput target validation assay that represents biology
- Molecular level
- Screen enzyme inhibitors or activators
- Cellular Level
- Verify the involvement of the protein in the disease state (often use gene silencing siRNAs)
- Understand the protein pathways and interactions
Target Validation - Gene knockdown
HeLa cells transfected with the siGLO™ transfector indicator (A) siNegative Control and siWRN. WRN gene expression was assessed 72 hours post-transfection by Western Blot (B) and Quantigene ™ assay (C) to confirm the siRNA knockdown
Target Validation - expression
Chemiluminescence imaging of basal levels of alpha Smooth Muscle Actin (α-SMA) expression over 72 hours in human lung fibroblasts and mouse lung fibroblasts by western blotÂ
                    Immunofluorescence staining of Hela cells expressing α-Smooth Muscle Actin
Purity of freshly isolated primary human neutrophils was analysed by FACS for surface markers (CD45 and CD66b). Data shows gating strategy for characterising the neutrophils. Neutrophils were stimulated with TNF-alpha for 4 hours. Differences in LFA-1 surface antigen expression between unstimulated (B.1) and stimulated (B.2) neutrophils was observed.
Request a consultation with Cellomatics Biosciences today
Our experienced team of in vitro laboratory scientists will work with you to understand your cell-based assay development needs and provide a bespoke project plan with a professional, flexible service and a fast turnaround time.
To request a consultation where we can discuss your exact requirements, please contact Cellomatics Biosciences.
FAQ
Target validation assays confirm that a biological target plays a functional role in a disease process or therapeutic response. They establish causality between target modulation and biological outcome.
They are typically applied after initial target identification to confirm relevance across multiple experimental conditions before advancing into drug development; at Cellomatics, this stage is supported through integrated functional and molecular assays, including genetic modulation approaches and human-relevant models, to strengthen target validation.
By demonstrating how target modulation affects cellular or molecular pathways, these assays provide evidence for mechanism of action and guide candidate selection; Cellomatics supports this through combined use of knock-out/knock-in, overexpression systems, and multi-parametric readouts to deliver robust, decision-enabling data.Â
Common approaches include genetic modulation (e.g., knockdown or overexpression), pharmacological inhibition, and pathway analysis. Cellomatics integrates these with functional and molecular readouts for comprehensive validation.
They link target activity to measurable biological changes, establishing cause-and-effect relationships that support further development.
By identifying targets with weak or inconsistent effects early, these assays prevent investment in non-viable strategies, improving overall success rates.