Target identification and validation

We offer a collaborative and robust experimental design approach and can guide on the most suitable cell types, in mono- or co-culture assays to validate your hypothesis. We also support de novo target validation, conducting studies in primary tissue to answer questions about key proteins and genes involved in your disease of interest.

We offer a wide range of in vitro and in vivo assays and design experiments with tailored readouts such as FACS, ELISA, multiplex and RNA-seq to suit your requirements. This translational approach can be used to develop assays that mimic the tumor immune cell environment (TME), a specific autoimmune condition or neurodegenerative disease, providing sophisticated in vitro human models to enable you to better predict the translation from animal models to activity in the clinic.

Questions we can help you answer

How do I identify potential therapeutic targets in my disease of interest?

We can support your understanding of disease pathology by providing analysis of matched tissue between diseased and non-diseased patients. We can interrogate pathways, genes and protein targets involved by performing expression profiling (e.g. taqman, IHC, Western, RNA-Seq, Nanostring) as well as developing novel custom primary cell and co-culture assays to explore your hypothesis.

How can I understand if the phenotype I need to inhibit is due to the enzyme activity or the scaffolding function of my protein?

We provide a Targeted protein degradation service. We can design PROTAC degraders to eliminate your protein of interest at the protein level and interrogate the phenotype of protein knockdown. We can also identify novel inhibitors via our unique biophysical fragment-based drug discovery platform.

What is my drug’s mode of action?

We design custom novel biophysical and biochemical assays to determine the binding mode and mechanism of protein-drug interaction. For example, we can interrogate whether your drug candidate prevents a protein complex associating, or stabilizes a protein complex in an inactive state thus inhibiting downstream events. We can also interrogate the kinetic profiles of these interactions. Or we can look at whether your compound is an activator, or inhibitor, (competitive, non-competitive), whether your binding mode is allosteric or orthosteric.

What are the downstream targets and biological consequences of the action of my drug?

We can support assay design in cell-based assays to provide analysis of the molecular signaling pathways impacting in different cell and tissue types.

Are the results I see in in vitro assays mirrored in vivo and in disease-relevant states?

We offer in vivo animal-based disease models in our therapeutic areas of expertise immunologyimmuno-oncology and neuroscience 

We also support these models with a range of ex vivo analyses such as brain slice models, multiplex histology, Nanostring and RNA-seq

How do I know that the results I see in my in vivo model will translate to the clinic?

We are experts in developing human in vitro cell-based co-culture disease models using primary cells, tissue (diseased and non-diseased) or cell lines. We also provide matched models and readouts in rodent in vitro models to support the translation of your in vivo efficacy studies to prediction of drug behavior in humans.

An output of this analysis is that we identify potential biomarkers and/or bioanalytical readouts suitable for monitoring outcomes in your clinical trial (eg qPCR, LCMS/MS, MSD, Flow cytometry, Histology, RNA-seq and ELISA readouts)

Want to find out more?

Our multi-disciplinary teams mitigate risk by employing knowledge, foresight and thought leadership to all our client’s diverse programs enabling the highest chance of success. Get in touch to find out how we can support you.