Purposeful is an in silico drug repurposing company, with a primary focus on rare diseases. Their company uses articifical intelligence in disease models to ascertain whether certain, already FDA approved drugs or nutraceuticals may be beneficial in a particular disease. Through this process Purposeful has already uncovered a handful of possibled drugs that could be efficacious in Pitt Hopkins, the most promising of which we are working to bring to trail by 2024. Their work can be summarixed in the following bullets.

● Small molecules. They have mined over 2M useful experimental molecules and have used our custom in-house algorithm for describing chemical space associated with the target/pathway level. These are used to make predictions for approved drugs/nutraceuticals.
● Diseases. All diseases for which sufficient information is present in the literature. They look for genetic associations, somatic mutations, known pathways, RNA-seq data, as well as text mining, effects of current drugs on the disorder/similar/pathway and the existing animal models.
● Filters. This node includes anything from setting biological activity thresholds to determine when a small molecule is considered active, likelihood cut-offs, method parameters, pediatric approvals, all the way to the cost of a clinical trial.
● Modelling. They use a consensus of 180M models which provide us with novel information on the candidate drug and then assess the significance of the new information for each disease with 120M statistical enrichment calculations.
● Post-discovery. Ranking of candidates based on current literature, patents, mechanism of action, drug category, promiscuity, risk-benefit ratio, and so on. At this stage it is common to reach out to world experts in the area, to assess next steps, commonly preclinical validation
● Validation and next steps. Confirmation in a suitable animal model, IP protection, regulatory aspects in order to move towards a pilot trial.