Target ID & Validation

To find and validate the right target for your indication, partner with us. We are experts at identifying and verifying targets for various therapeutic indications. We build tailored models with specific readouts and apply technologies, such as bioinformatics, bulk and single-cell next-generation sequencing, RNA interference, CRISPR/Cas9, and chemical probes. Furthermore, target expression in relevant tissues and cells is evaluated from clinical samples. We can also provide early insights into the mode of action and conduct assayability (àbelow) and druggability (àbelow) assessments to support your project.

In Silico, in Vitro, and Functional Genomics-Based Target Validation

We combine refined bioinformatics approaches and functional genomics to identify attractive drug targets. We offer pooled and arrayed CRISPR screens with customized readouts in scalable formats up to genome-wide screens. Target validation is supported by tailored cell-model development (cell lines, iPSC-derived cells, and primary cells in 2D, 3D, and co-culture formats). Our biosafety level 2 laboratories enable lentivirus- or AAV-mediated transgene delivery. CRISPR knock-out or knock-in studies are complemented by RNAi. We use our internal next-generation sequencing to investigate, for example, RNA expression, chromatin modifications, or DNA mutations. In addition, our 10x Genomics single-cell sequencing platform offers high-resolution gene expression analysis, B- and T-cell receptor profiling, and CITE- and ATAC-seq of preclinical and clinical samples.

Biomarker Hypothesis Generation

After a target is identified, early insights into the mode of action of the target help to de-risk projects by having a focus of on-target activity. We identify target engagement/pharmacodynamic biomarkers, combining experimental approaches with in-depth bioinformatics and literature studies.

Assayability—Assay Feasibility Assessment

To minimize risks to your project, we support you with a feasibility evaluation of the assay system, which is a crucial step before starting the actual assay development. Through targeted research, the different assay options are evaluated and ranked. Our assessment informs you about assay stability and reproducibility, sensitivity, miniaturization, and suitability for use in an automated industrial laboratory to save you costs and increase efficiency.

Druggability Assessment

Also crucial is the druggability assessment of your target. Selective binding of small-molecule ligands to biological target structures requires particular features to be considered before the initiation of a drug discovery campaign. We have many years of experience in analyzing targets for feasibility of structure determination, the existence and suitability of binding pockets, the likelihood of induced-fit events, and potential off-targets to be included in a screening campaign.

Indication Space Assessment

In case you already have a compound of interest, we support you in assessing the indication space using big data analysis. Our 10 years of expertise in positioning, indication expansion, repositioning, and repurposing can help identify the most suitable proof-of-concept indication for your asset. We propose feasible paths forward by identifying the most translational in vivo or ex vivo models and have a track record of successfully finding indications in unexpected areas. In silico, in vivo, ex vivo, and in vitro studies can be performed in our labs or in coordination with third-party partners. From conceptual drafting to clinical translation and regulatory documentation, we provide support by de-risking every step of your project.

Our target ID and validation solutions cover: