Abstract
Establishing effective therapies for rare neurodevelopmental diseases remains one of the greatest challenges in molecular medicine. Although advances in next-generation sequencing technologies have led to the discovery of hundreds of novel genetic syndromes over the past decade, the development of individualized therapies continues to lag behind. Each rare disorder, while affecting a small group, contributes to a global burden estimated to impact over 300 million individuals. The complexity arises from the fact that these disorders, often caused by mutations in different genes, affect multiple cellular pathways, generating an overwhelming volume of data that must be analyzed to inform therapeutic strategies. Current drug interventions have seen limited success in translating promising preclinical findings into patient-ready treatments.
The rapid rise of AI technologies, however, has the potential to transform this landscape. AI-driven algorithms are increasingly capable of navigating vast biomedical datasets, revealing drug candidates for rare diseases at an unprecedented pace. Many start-ups are already capitalizing on this potential, generating a flood of drug candidates for preclinical evaluation. However, this surge in candidate therapies has shifted the bottleneck from drug discovery to preclinical testing. Traditional murine test batteries are labor-intensive, expensive, and time-consuming, necessitating a standardized, scalable, and efficient platform to meet the growing demand for drug screening.
We propose the development and commercialization of our Live Mouse Tracker (LMT) platform, a cutting-edge tool designed to address this critical need. The LMT system automates behavioral analysis, capable of tracking up to 39 different behaviors in groups of mice over 24-hour periods. This high-throughput capability provides a rapid and comprehensive assessment of drug efficacy in preclinical models. Our initial validation will focus on the fragile X syndrome, a widely studied neurodevelopmental disorder for which no effective treatment currently exists. By evaluating drugs that target multiple affected pathways simultaneously, we aim to pioneer a new approach to rare disease therapy development.
During this project, we will validate the robustness of the LMT platform and extend it into a fully integrated service, as well as explore collaboration with other university partners to offer comprehensive preclinical drug testing solutions. This service platform has the potential to revolutionize the drug development pipeline, ensuring that AI-generated candidate drugs can be rapidly and reliably assessed, accelerating the path from bench to bedside. Through this initiative, we aim to bridge the gap between drug discovery and therapeutic application, bringing hope to millions of patients with rare neurological diseases.
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