Fulcrum Therapeutics - Company Profile

Fulcrum Therapeutics is focused on discovering and developing small molecules that modulate the on/off control mechanisms that regulate genes. The Fulcrum product engine integrates insights from four scientific disciplines (Gene Regulation, Human Genetics and Disease, Cell Biology, and Screening and Computation) to identify novel drug targets and develop breakthrough medicines. The early focus of Fulcrum’s product engine is on two severe genetic diseases: Fragile X syndrome (FXS) and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD). Each disease arises from a single gene mutation that creates an error in gene regulation. In the case of Fragile X, the silencing of the FMR1 gene eliminates the cell’s ability to make a protein needed for brain function. In FSHD, the precipitating event is the activation of a gene called DUX4 that should be silent in adulthood. This activation leads to muscle wasting as cells die.