Advancements in artificial intelligence are revolutionizing drug design, particularly in central nervous system (CNS) research, according to David Del Bourgo, CEO of WhiteLab Genomics.
Del Bourgo emphasizes a shift from traditional trial-and-error methods in drug development to a more engineered approach. By utilizing AI, WhiteLab identifies specific receptors unique to target cell types, ensuring these receptors are present on the cell surface. Their proprietary mapping technology leverages human data to pinpoint these targets, which are crucial for developing effective genomic medicines. An essential aspect of their process is an algorithm that assesses receptor conservation across species, allowing for initial testing in animal models like mice and primates.
Once a target receptor is identified, the next step involves attaching it to a vector without compromising its functionality. WhiteLab employs both viral and non-viral vectors, including adeno-associated viruses (AAVs), to enhance gene therapy applications. Del Bourgo notes significant progress in their CNS-focused project, where they have engineered a new vector that surpasses traditional AAV9 in performance, particularly in its ability to cross the blood-brain barrier. Remarkably, this new vector shows negligible liver accumulation, a significant improvement over existing options.
The company's AI-driven approach has yielded impressive results, achieving a 4% hit rate from 15,000 candidates, with 600 viable leads. Del Bourgo indicates that they are now preparing for in vivo studies in larger animal models and are in discussions with various pharmaceutical partners. This innovative use of AI not only accelerates drug design but also aims to mitigate risks associated with genomic medicine development, heralding a new era in CNS research.