Therapeutic Focus

Patient-informed discovery and patient-driven development

Patients are central to our mission at Fulcrum. Partnering with the patient community is also essential to our process. Our drug discovery begins with a deep exploration of disease biology in patient tissue samples. As we advance a program, the patient voice guides our development strategy.

Our initial areas of focus are facioscapulohumeral muscular dystrophy (FSHD), sickle cell disease (SCD), and non-SCD hemoglobinopathies, including beta-thalassemia. We’re developing precision medicines that address the root causes of these genetic conditions.