Third Rock-backed Fulcrum Therapeutics launches

Like all Third Rock-backed companies, Fulcrum gestated inside the venture firm’s labs for several years before making its debut last week. The biotech’s discovery engine is based on several emerging areas of science: developing safe and selective small molecules to modify gene expression, generating induced pluripotent stem (iPS) cells from patient skin cells, and using the gene editing tool CRISPR to validate drug targets.

Fulcrum will first go after two rare diseases that lack treatments. For the cognitive disorder fragile X syndrome, the company will try to activate the production of FMRP protein. For fascioscapularhumeral muscular dystrophy, the goal is to prevent muscle cell death by turning off expression of the DUX4 gene.

Fulcrum launched with 10 employees, a number that will grow to about 40 by the end of 2017, says CEO Robert Gould, who previously led the small-molecule epigenetics firm Epizyme. Most of those hires will be scientists, including experts in iPS cell biology, bioinformatics and computational biology, and translational medicine.

Researchers in that last area—who are moving discoveries out of the lab and into the clinic—are being brought on early in acknowledgment of the challenge of designing trials for rare neurological and muscle disorders. “The translational medicine piece is really critical,” Gould says. For example, a study on a disease-modifying drug for fragile X has never been done; Fulcrum will need to figure out the right questions to ask.