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.