The Genetic, Rare, and Immune Disorders Symposium (GRIDS), an annual Continuing Medical Education (CME) conference organized by the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), provides a dedicated platform for clinicians, researchers, and industry leaders to discuss and disseminate the latest breakthroughs in LDs. GRIDS2025, scheduled for November 16-17, 2025, at Capital One Center, Tyson’s Corner, VA, will focus on these groundbreaking advancements, including CRISPR-based diagnostic technologies, novel imaging methods, nanomolecule-mediated drug delivery, and 3D disease modeling techniques.

Over the past decade, tremendous progress has been made in understanding lysosomal biology, leading to novel therapeutic innovations, including enzyme replacement therapy (ERT), substrate reduction therapy (SRT), gene therapy, and RNA-based treatments. However, despite these advancements, significant knowledge gaps and treatment limitations persist, especially in addressing neurological involvement and long-term disease progression.

Recent advances in nanotechnology, gene therapy, and disease modeling are revolutionizing the LD research and therapy landscape. Nanomolecules, including nanoparticles, liposomes, and exosomes, offer novel drug delivery mechanisms, enabling targeted therapies with enhanced precision and reduced systemic toxicity. CRISPR-based diagnostic platforms are emerging as powerful tools for early detection. In contrast, advanced imaging techniques utilizing lysosomal probes and fluorescence-based detection methods provide unprecedented insights into lysosomal function and disease progression.

Additionally, 3D organoid and spheroid models derived from patient cells provide more physiologically relevant platforms for drug screening and pathophysiological studies, bridging the gap between preclinical models and human trials. These advances, combined with multi-omics technologies and AI-driven computational analysis, propel the field toward personalized and precision medicine approaches for LDs.