AI Workbench
We use our AI Workbench to rapidly discover and develop genetic medicines, and to do so with a high rate of success

Steric Blocking Oligonucleotide (SBO) Therapies

Our AI Workbench enables us to efficiently find drug candidates with desirable properties. We are focusing on the development and marketing of steric blocking oligonucleotides (SBOs) 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.

Our Platform is Getting Better, Faster

We collect data on every compound identified using our platform. That includes therapeutic candidates and thousands of novel exploratory compounds. We also collect data pertinent to biomarkers 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.

World Class Science

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.

AI Workbench 3.0

Through iteration and positive feedback loops, prediction with our AI Workbench improves and unlocks new therapeutic opportunities.

Our AI Workbench 1.0 focused on correcting RNA splicing as a means to restore protein expression. We recognized that a wide range of disorders, such as haploinsufficiencies can be potentially treated by boosting gene expression. Version 2.0 of the workbench was expanded to include seven mechanisms to increase expression and reduce the work needed to identify efficacious compounds. We are currently developing AI Workbench 3.0 to support target identification and drug discovery for more common, complex diseases involving multiple genes.We are now advancing a pipeline of programs in a number of areas, including neurodevelopmental, neurodegenerative and metabolic.

Publications

Our scientists regularly publish in their fields' most influential peer-reviewed journals. Here is a selection of our contributions.