Target Identification & Validation

Based on our long-term pharmaceutical research experience we can provide you with knowledge on cutting edge drug development, innovative research practice and how to advance your project optimally towards your goal. We have experts in all fields of the non-clinical and early clinical value chain - from hypothesis generation to lead structure identification and optimization, from DMPK to pharmacology, from in vitro to in vivo, from model to patient. We pave the way for your compound's clinical success in the most attractive indications.

We support you to find the most suitable disease fit to your compound.
Applying in silico biology tools, big data mining, database screening, extraction and assembly, we identify pathway-disease-relationships and entity recognitions. We scrutinize your target and it’s signaling pathways, and the Mode of Action of your compound. We help you to identify pathomechanisms that offer therapeutic rationales with optimum fit to your asset.

Combining refined bioinformatics approaches and functional genomics, we identify attractive drug targets. We offer pooled and arrayed CRISPR screens with customized readouts in scalable formats up to genome-wide screens. Target Identification is supported by tailored cell model development as well as next generation sequencing to investigate e.g. chromatin modifications, DNA mutations and RNA expression. In addition, our 10x Genomics single cell sequencing platform offers high resolution gene expression analysis and ATAC-Seq.

We will help you in finding and validating the right target for your indication

We have extensive experience in target identification and validation across a broad spectrum of therapeutic indications. NUVISAN’s scientists have access to state of the art technologies, including bioinformatics, single-cell next generation sequencing, RNA interference, and chemical probes in selected model systems. Our CRISPR expertise empowers functional studies from the investigation of individual genes to genome-wide phenotypic screens for target identification or deconvolution. This is complemented by profiling of clinical sample collections for target expression in relevant tissues and cell types.

We identify the most suited indication to develop your asset

We map the specific indication space for your asset using big data analysis tools. We propose first positioning, indication expansion, repositioning, or repurposing building based on our 10 years plus of experience. We identify the most suited proof-of-concept indication for your asset. We propose feasible paths forward by identifying the most translational in vivo or ex vivo models. We have a track record of projects that found their home in often unexpected indications. We perform in silico-, in vivo-, ex vivo-, and in vitro-studies for you in our labs - or we orchestrate your studies with third partners. We assist you from conceptual drafting to clinical translation and regulatory documentation by de-risking every step.

NUVISAN can help you to get early insights into the Mode of Action of the target, thus helping to de-risk projects by focusing on on-target activity. We can support you with the identification of Target engagement and Pharmacodynamic biomarkers based on in-depth studies of the target biology using literature searches as well as bioinformatic analyses. .

How druggable is your target of interest?

Selective binding of small-molecule ligands to biological target structures requires features to be considered before the initiation of a drug discovery campaign. We have many years of experience in analyzing targets regarding the following questions:

• Is there a reliable basis to assess the structure of the target of interest?
• Are there interaction pockets embedded within the target of interest?
• Are there potential allosteric pockets?
• Are these pockets suitable for lead-like ligands or fragments?
• How do lipophilicity and electrostatic contributions in and near the binding site behave?
• Are there induced-fit events observed or likely to occur?

What are the closest structural neighbours being potential off-targets, to be included into selectivity panels or counter screens, to monitor potential unwanted cross-reactivity?

The feasibility evaluation of the assay system is a crucial step before starting the actual assay development. Through targeted research, the different assay options are evaluated, compared and ranked. With our many years of experience in industrial laboratory practice and pharmaceutical research, we support you in the selection of the most suitable system for answering your research questions. At its core, our feasibility study consists of the following questions: Which assay approach provides the best-suited model? Which assay approach provides the most stable and best reproducible data? Which assay approach is the most sensitive? Which assay approach can best be used in an automated industrial laboratory? Which assay approach is best suited for miniaturization to save costs and test as many samples as possible in a particularly short time?

We have extensive experience in target identification and validation across a broad spectrum of therapeutic indications. We use state of the art technologies including bioinformatics, RNAi, CrispR gene editing and chemical probes, along with profiling of clinical sample collections for target expression in relevant cell types which are available to help support your project.