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 beta thalassemia. We’re developing precision medicines that address the root causes of these genetic conditions.