Quiver Bioscience use functional genomics to unravel the complex inner workings of the brain for drug development. Graham Dempsey's presentation provided an insightful overview of the challenges and innovations in drug development for brain disorders. He began by highlighting the historical difficulties in developing effective treatments for brain disorders due to the complexity of disease biology and the challenges faced in phase two clinical studies.
Dempsey explained that the brain's electrical activity is fundamental to understanding how we think, sense, and experience disease. However, measuring these electrical signals has traditionally been difficult and not scalable for drug discovery programmes. To address this, Quiver Bioscience has developed a functional genomics platform that translates brain electrical signalling into machine-readable signals. This platform uses patient-derived brain cells and converts electrical activity into detectable light signals, enabling high-throughput measurement and analysis.
The platform leverages optogenetics, expressing proteins in neurons to stimulate and read out activity using light. This approach allows for the creation of high-dimensional data sets that could be analysed using machine learning to identify patterns in health and disease. Dempsey emphasised how functional genomics can link changes in gene expression to disease outcomes, ultimately bridging the gap between target mechanisms and clinical phenotypes.
He provided examples of the platform's application in studying diseases such as fragile X syndrome and tuberous sclerosis. By creating human neuronal reagents and using CRISPR to disrupt genes, the platform identified phenotypes and screened for therapeutic compounds. The platform's scalability was demonstrated through its ability to measure from millions of neurons in high-throughput formats, producing complex but informative data sets for drug discovery.
Dempsey concluded by discussing Quiver Bioscience's future goals, including scaling the approach to build phenotypes for hundreds of diseases and leveraging CRISPR genomic screening to create a comprehensive knowledge graph of neuronal biology. He expressed confidence in the platform's potential to revolutionise drug development for brain disorders.
