Core Insights - Polaryx Therapeutics is committed to developing disease-modifying therapies for rare pediatric lysosomal storage disorders (LSDs) and raising awareness within the rare disease community [2][4] Company Overview - Polaryx Therapeutics, founded in 2014, focuses on small molecule and gene therapy treatments for rare orphan LSDs, aiming to address significant unmet medical needs [8] - The company's lead candidate, PLX-200, is designed to target multiple LSDs and is currently advancing through clinical trials [8] Clinical Trials - The SOTERIA trial is a Phase 2, open-label, single-arm study aimed at evaluating the safety, tolerability, and clinical activity of PLX-200 across four rare LSDs: CLN2, CLN3, Krabbe disease, and Sandhoff disease [7] - The trial is expected to begin in the first half of 2026, with sites in the United States, Europe, and Asia [7] - SOTERIA is designed to be flexible and resource-efficient, potentially validating PLX-200's preclinical science while gathering important data for future clinical development [7] Rare Disease Context - LSDs are a group of over 50 inherited metabolic diseases caused by genetic mutations, affecting approximately 1 in 5,000 births [3][4] - The National Organization for Rare Diseases (NORD) defines a rare disease as one affecting fewer than 1 in 2,000 people globally, with about 70% starting in childhood [4] - More than 300 million people worldwide are affected by rare diseases, highlighting the urgent need for new treatments [4]