At Fulcrum – above all else – our goal is to make a positive impact in the lives of patients and families impacted by severe disease.
For each of its lead programs, Fulcrum is gratified to be working closely with patient groups who are partners in our search to better understand the disorders and to develop breakthrough medicines. For every program the company starts, we regard partnership with patient groups as essential to our work.
Fulcrum Therapeutics Pipeline
In all of us, genes are regulated through a balance of active and repressive states. There are hundreds of serious human disorders for which a controlled change in the expression of a particular gene might significantly ameliorate disease. By unlocking the mechanisms of gene regulation, Fulcrum’s product engine represents an opportunity to develop disease-modifying therapies for many diseases across therapeutic areas: neurodevelopmental, neuromuscular, neurodegenerative and many others.
Fulcrum’s unique and differentiated approach addresses the genetic cause of disease. In our initial efforts, we will be focusing on monogenic diseases that arise due to a faulty regulation of a single gene.
Sponsored Research Studies
Fulcrum is committed to working with academic scientists, key thought leaders, and patients to identify disease-relevant biomarkers, and to define clinically meaningful outcomes. We intend to rapidly and efficiently transition our discoveries into clinical development strategies. Learn more about these studies here: