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. At Fulcrum, we are driven by a commitment to make a difference for patients with genetically defined diseases. We have been privileged to spend time with many of these patients and their families. Their experiences guide us as we work to develop therapies that will lead to meaningful improvements in their day-to-day lives. Read more about the patients who inspire us.
