Fulcrum Therapeutics pioneers a small-molecule, precision-medicine approach to modulate gene regulation and address monogenic and other genetic diseases. Its core programs target disease-causative genes to unlock druggable mechanisms and create new therapies across therapeutic areas. The company’s lead program is focused on facioscapulohumeral muscular dystrophy (FSHD) and is being advanced toward clinical testing. Fulcrum has initiated clinical trial readiness studies in FSHD in partnership with the FSHD Clinical Trial Research Network to standardize tools and measurements for future trials. Proceeds from the recent financing will support moving the lead candidate into the clinic and progressing a pipeline targeting rare neuromuscular, central nervous system, and hematologic disorders. Fulcrum is headquartered in Cambridge, Mass., and was launched by Third Rock Ventures in 2016. Fulcrum Therapeutics discovers and develops small-molecule therapies that modulate gene regulatory switches to treat serious genetic diseases. Its lead programs target Fragile X syndrome (FXS) and Facioscapulohumeral muscular dystrophy (FSHD). The company says its approach focuses on gene regulation pathways rather than the genes themselves. Fulcrum was founded in July 2016 and was launched by Third Rock Ventures. It is headquartered in Cambridge, Mass. The company announced additional Series A funding from GV to support continued advancement of its lead programs and broader goal of bringing new therapies to patients and families. Fulcrum Therapeutics was launched in 2016 to discover and develop small-molecule therapies that modulate gene control mechanisms governing when disease genes are turned on or off. The company built a cross-disciplinary product engine that integrates transcription biology, stem cell biology, computational biology, patient-derived cells (including iPSCs), CRISPR/CAS9 screening and chemical probe libraries, plus public gene-regulatory datasets to map and prioritize targets. Its early pipeline is led by two initial programs targeting Fragile X syndrome (FMR1 silencing) and facioscapulohumeral muscular dystrophy (DUX4 activation), with plans to expand to other neurologic, skeletal muscle and cardiac diseases. Fulcrum intends to use its genome-wide maps of gene regulation to rapidly identify drug targets for activation or repression of disease genes. The company is led by CEO Robert J. Gould and was launched by Third Rock Ventures. Fulcrum is headquartered in Cambridge, Mass., and was established with a $55 million Series A financing to build out its discovery and development capabilities.

Total raised

$135M

Funding rounds

3

Latest round

Series B

Latest activity

Sep 2018