GM2 gangliosidosis and Krabbe disease
Polaryx Therapeutics is finishing getting ready for the start of the SOTERIA Phase 2 basket trial which they aim to initiate in the fourth quarter of 2026. SOTERIA is a Phase 2, open-label, single arm trial where all participants receive the same drug. It is designed to evaluate the safety, tolerability, pharmacokinetics (how the body processes a drug) and clinical activity of the lead candidate drug PLX-200 in paediatric patients with CLN2 disease, CLN3 disease, Krabbe disease and Sandhoff disease. They have registered on ClinicalTrials.gov under the study identifier NCT07740512 which will provide important information including eligibility and active sites as they become available.