Research & Development
Novel, human genetic-based discoveries
Developing new treatments for patients is challenging, with just one in ten drugs entering clinical trials being approved. However, drugs supported by human genetic evidence are over twice as likely to succeed in clinical trials, making it critical for modern drug discovery. We leverage unique data from our genomic partnerships around the world to identify first-in-class targets with strong genetic support that can rapidly progress to the clinic and provide life-saving therapies to patients.
Human genetic validation is the foundation of all our programs
Multi-omic data are integrated using the VB-Inference platform to identify causal genes and establish directionality so that clear therapeutic hypotheses can be formed. Targets with converging genetic evidence across cohorts and ancestries are prioritized. Before initiating drug discovery, we validate therapeutic hypotheses using a combination of in vitro and in vivo models with strong links to disease-relevant mechanisms. We advance programs that are druggable by standard modalities and have existing tool compounds, or where our novel genetic insights identify in-licensing opportunities for clinical stage assets.
Our approach to research
Our R&D team includes industry-experienced biologists and chemists with track records of success in producing clinical stage molecules (both small molecules and biologics). Capabilities encompass: molecular biology, biochemistry, cell biology, pharmacology, medicinal chemistry, analytical chemistry, drug metabolism, and pharmacokinetics.