Our AI Workbench enables us to efficiently find drug candidates with desirable properties. We are focussing on the development and marketing of oligonucleotide therapies that target the genetic determinants of disease at the level of RNA or DNA. These genetic diseases are mediated by altered molecular phenotypes, such as transcription, splicing, translation and protein binding. Predicting those alterations is our core competency. The oligonucleotide therapeutic design space includes tens of billions of compounds, but our platform makes it possible to search this space efficiently.

On-target and genome-wide off-target effect data, cell viability data and animal toxicity data are produced for every compound identified using our platform. That includes therapeutic candidates and thousands of novel exploratory compounds. We also collect data pertinent to biomarkers that are used as endpoints in our clinical trials. All of that data is fed back into our AI system, closing the loop. We even test compounds that push the boundaries of what’s possible. This means that our platform is getting better at identifying extraordinary targets and compounds, and is doing so faster.

Our platform incorporates the most advanced biological and medical knowledge, is driven by the most powerful automation technologies, and is built using proprietary as well as public datasets. Our work has appeared in Science, Nature, Nature Genetics, Nature Medicine, Nature Methods, Proceedings of the IEEE, NIPS, Bioinformatics, RECOMB and ISMB. When other scientists and engineers publish a discovery, we evaluate it and if appropriate rapidly incorporate it into our platform.