NanoDiscs for Immune Modulation in Celiac Disease

PROJECT SUMMARY/ABSTRACT Celiac disease (CeD) affects approximately 1% of the global population, with gluten exposure leading to symptoms such as nausea, vomiting, and damage to the small intestine. Although a lifelong gluten-free diet is the current standard of care, strict adherence is challenging, and disease symptoms may persist despite compliance. This underscores the urgent need for novel therapeutic strategies. We are developing an interdisciplinary bioengineered platform focused on nanotechnology for targeted drug delivery in CeD. Our research emphasizes the formulation, manufacturing, and in vivo investigation of synthetic high-density lipoprotein NanoDiscs. These NanoDiscs are designed for controlled release of peptide antigens directly to lymph nodes. In preclinical studies, NanoDiscs have demonstrated strong potential for promoting immune tolerance in various murine models, including CeD. Additionally, our pre-IND meeting with the FDA has provided valuable feedback on our manufacturing plans, clinical trial design for CeD, and the required preclinical dataset for IND submission, guiding our research strategy. Building on these promising preclinical results and regulatory insights, we propose to advance NanoDiscs as a novel therapeutic solution for CeD. By leveraging biomaterials, nanotechnology, and advanced drug delivery systems, our research aims to address the unmet needs in CeD treatment and provide patients with more effective therapeutic options. This approach could also pave the way for new strategies in treating other conditions requiring targeted delivery of therapeutic agents.