Finding effective treatments for Fragile X syndrome remains one of the biggest challenges in translational medicine. While scientists have identified many biological pathways affected in Fragile X syndrome and have found promising therapies in animal models, translating these findings into patient-ready therapies has proven difficult.

Artificial intelligence (AI) shows massive potential to transform this landscape, by rapidly scanning massive biomedical datasets to uncover new drug candidates for Fragile X syndrome. But identifying potential treatments is only the first step. The real challenge lies in testing them quickly and reliably, shifting the bottleneck from drug discovery to preclinical testing. Traditional behavioral testing in mice is labor intensive, time consuming, and non-standardized highlighting the urgent need for a standardized, scalable and efficient for preclinical drug screening platform.

To overcome this we use the Live Mouse Tracker (LMT) platform. This system automatically monitors up to 40 behaviors in groups of mice over 24 hours, capturing detailed patterns in activity, social behavior, and anxiety. Together with our in-house developed MouseKing data analysis pipeline we have developed a versatile, innovative, and efficient platform for drug screening in Fragile X syndrome.

Our platform is the first universal system capable of testing multiple drugs under identical and standardized conditions. This highly innovative project builds on our previous FRAXA grant and integrates next-generation mouse phenotyping, AI-driven drug discovery, and transcriptomic profiling to identify novel therapeutics for Fragile X syndrome.