Our key small molecule capabilities include:

Target Validation:
We apply rigorous hypothesis testing using cutting-edge knockout strategies, including CRISPR/mad7, to validate therapeutic targets. Our in vitro systems span a range of complexities, from simple signalling reporter assays to sophisticated patient-derived organoid models, ensuring a thorough examination of biological functions. Additionally, we perform expression analysis in both diseased and healthy donors, providing robust data for target confirmation.

Extensive Binding Characterisation (SPR):
RoukenBio has deep expertise in Surface Plasmon Resonance (SPR),  offering precise binding characterisation of small molecules. We specialise in building complex binding models tailored for binary and ternary complexes, which are critical for understanding the functionality of PROTAC molecules.

Biology Assay Development:
Developing robust biological systems is a major hurdle in small molecule discovery. At RoukenBio, we utilise advanced cell engineering capabilities to create highly reliable cell-based systems for interrogating diverse biological functions. As experts in bioanalytical assay development, we design reproducible, accurate, and precise assay systems that are optimised for high-throughput screening and hit-to-lead transitions, ensuring data integrity throughout the discovery process.

Lead characterisation and optimisation:
Our advanced in vitro models provide deep characterisation of the efficacy, selectivity, and potency of lead candidates. By generating structure-activity relationship (SAR) data and performing mechanism of action (MOA) characterisation using high-content platforms, we support the development of comprehensive in vitro pharmacology data packs to streamline clinical translation.

Disease Modelling for Candidate Characterisation:
RoukenBio excels in establishing disease-relevant models using patient-derived material, 2D and 3D multicellular systems to replicate the intended clinical use of your therapeutic. We perform early biomarker assessments and, as immunology experts, interrogate molecular impacts across a range of pathological settings, including autoimmune diseases, atopic conditions, and immuno-oncology. With access to healthy and disease-specific donor material from our well-characterised donor bank, we provide consistent and reliable data to support your drug development efforts.

Preliminary Immunotoxicology:
For therapeutics that interact with the immune system, we assess potential risks of unwanted immune activation, focusing on cytokine release syndrome - a life-threatening immune response. Using large donor cohorts and high-content cytokine analysis, we deliver critical immunotoxicology data to ensure the safety of your candidates before clinical trials.
 

By leveraging our expertise in target validation, translational biology, and immunology, RoukenBio provides a comprehensive suite of capabilities to support every stage of small molecule development, driving your projects from discovery to clinical success.