12:00 AM
Jul 25, 2016
 |  BioCentury  |  Finance

Leverage over genes

Why Third Rock, Fulcrum say the time is ripe for a broader approach to gene control

The decision to invest $55 million in a series A round for Fulcrum Therapeutics Inc. reflects Third Rock Ventures' belief that research tools including induced pluripotent stem cells, CRISPR-Cas9 gene editing and bioinformatics have matured enough to identify and screen small molecules that fix misregulated protein expression.

The newco hopes to have candidates ready for the clinic in two years, starting with fragile X and facioscapulohumeral muscular dystrophy. Both are caused by single mutations in...

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