Agora Open Science Trust is a Canadian charity whose mission is to accelerate the discovery and development of affordable new medicines through open science. Agora’s first initiative – M4K Pharma (‘Medicines for Kids’) – is using open science to drive preclinical and clinical development of a novel ALK2 inhibitor for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare pediatric brain cancer. Agora’s pipeline of collaborative open science drug discovery programs has recently expanded to include programs for Spinal Bulbar Muscular Atrophy (SBMA), a rare genetic neuromuscular disorder, and Primary Sclerosing Cholangitis (PSC), a rare liver disease—both of which currently have no approved treatment.