UAB Center for Precision Animal Modeling (CPAM) - Preclinical and Co-Clinical Section

ABSTRACT (PRECLINICAL AND CO-CLINICAL SECTION) The Preclinical/Co-clinical Section (PCS) of the Center for Precision Animal Modeling (CPAM) serves as a critical liaison between nominators—including patients, clinicians, and researchers—and the broader CPAM pipeline. Building on significant advancements from the previous funding period (CPAM1.0), where we streamlined the nomination and consent processes and established a secure data management system for 169 nominations, we propose to further integrate and advance PCS capabilities within CPAM through two specific aims. We will develop the HIPAA-compliant tool ASSIST (Artificial Intelligence-based Submission Support and Information Structuring Tool) to increase the quality and throughput of nominations (1a) and enhance the depth of clinical data extraction (1b). Leveraging artificial intelligence (AI) and large language models (LLMs), ASSIST will provide interactive guidance to nominators, ensuring submissions are complete and adhere to guidelines. It will also automate the extraction and structuring of complex clinical data from patient records. This innovation addresses current inefficiencies, reduces manual workload, and ensures high-quality data (i.e., data that meets clinical quality standards and metrics) for model development. Our second aim is to enhance the translational impact of CPAM, for which we will deliver a Therapeutic Analysis report (2a) and a Phenotype Validation and Clinical Curation report (2b) with each generated model. These reports will provide actionable insights, including potential therapeutic strategies and refined variant classifications based on model data. By collaborating closely with the Bioinformatics Section (BIS), Disease Modeling Unit (DMU), and Phenotype Validation and Clinical Curation (PVCC) Committee, we aim to bridge the gap between preclinical findings and clinical application, thereby accelerating therapeutic development. Our proposal addresses critical gaps by integrating co-clinical processes, establishing a model validation framework, and strengthening community engagement. By advancing PCS capabilities through these aims, we strive to accelerate the development of precise and impactful animal models, ultimately improving diagnostic precision and expanding therapeutic options for patients with rare genetic diseases. This work aligns with CPAM's mission to advance precision medicine and contributes to the broader goal of translating preclinical research into meaningful clinical outcomes.