Project: Drug Prediction using RNA from Human Nasal Swabs

Lead Investigator: Unravel Biosciences

Unravel Biosciences is the first rapid prototyping therapeutics company, integrating AI systems biology computation with rapid in vivo screening and clinical validation of discovered targets with unprecedented efficiency. Unravel leverages its proprietary BioNAV™ platform combining target and drug discovery, preclinical screening and patient stratification to find treatments for complex diseases. Unravel's platform has led to four clinical trials starting in 2024. Unravel's platform developed RVL002, a first-in-class new small molecule targeting mitochondrial metabolism, and RVL027, a molecule targeting a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform access to foundation and biotech partners to accelerate and clinically derisk therapeutics. unravel.bio and rareshift.org

Grant Details:

Project funded for 1 year starting in 2024 for a total of $43,000.

Aims of Grant:

Unravel will collect nasal swabs from 10 families (swabs from individuals with OCNDS and a sex-matched relative control, such as a parent or sibling). They will generate RNA sequencing data from the nasal swabs that will be used to predict drugs and therapeutic mechanisms using the BioNAV™ platform. Resulting predictions will be compared against results of the mouse drug prediction task to further narrow the predicted drugs for subsequent screening. This will generate the first primary clinical molecular dataset for CSNK2A1. For this pilot grant, individuals with the most common mutation in CSNK2A1 (K198R) will be analyzed.