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Genetic Disease Biotech Gains $55M in Early Funds

Genomics graphic

(National Human Genome Research Institute, NIH)

19 July 2016. A biotechnology start-up developing therapies for genetic diseases through regulation of genetic processes is raising $55 million in its first venture funding round. Fulcrum Therapeutics in Cambridge, Massachusetts is being established and initially financed by Third Rock Ventures, a venture capital company in Boston backing new scientifically-based enterprises.

Fulcrum Therapeutics is integrating technologies that regulate gene expression and transcription with computational biology to discover therapies at first for stubborn inherited diseases. The company’s initial targets are Fragile X syndrome and facioscapulohumeral muscular dystrophy, or FSHD. Each disease arises from a mutation resulting in errors in the regulation or expression of critical genes.

Fragile X syndrome affects learning and cognitive development in children, mainly boys, affecting speech and language, and resulting in symptoms similar to attention deficit disorder and autism spectrum disorder, as well as seizures. In Fragile X syndrome, a mutation silences the FMR1 gene that instructs the production of proteins needed for development of synapses, the signaling elements of nerve cells.

FSHD is a neuromuscular disease resulting in progressive weakness and atrophy of facial and shoulder muscles, usually in older children and teens. Children with FSHD often have difficulty sipping drinks through a straw, eyes that do not close completely, asymmetric shoulder development, and difficulty raising arms above the shoulder. In FSHD a mutation activates the DUX4 gene that should be silenced, which is believed to influence activity of other genes affecting muscle development.

Fulcrum Therapeutics expects to discover and develop small molecule, or low molecular weight, therapies affecting the on/off genetic switches for disorders like Fragile X syndrome and FSHD. The company plans to tap into advances in cell biology, including induced pluripotent stem cells, combined with concepts of precision medicine from genetics, as well as high-volume computational techniques to screen big data sets and identify drug targets.

The company says its technology reflects advances in genetics and medicine from its scientific founders:

Bradley Bernstein, professor of pathology, and director of the Epigenomics Program at the Broad Institute of MIT and Harvard

Michael Green, professor of molecular medicine at University of Massachusetts Medical School, and director of the school’s cancer center

Rudolf Jaenisch, professor of biology, and founding member of the Whitehead Institute for Biomedical Research at MIT, who works with stem cells and epigenetics

Jeannie Lee, professor of genetics and pathology at Harvard Medical School and molecular biologist at Massachusetts General Hospital

Danny Reinberg, professor of biochemistry and molecular pharmacology at New York University medical school

“One of the single most important biologic breakthroughs of the last decade has been the unraveling of gene regulation at a molecular level” says Fulcrum CEO Robert Gould in a company statement. “The richness of the human genome comes from the way genes are turned on and off. Through the modulation of these on and off switches we have the potential to transform the treatment of hundreds of serious human diseases.”

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