Black Rock Ventures is funding start - up Fulcrum Therapeutics with funding of $ 55 m to study genetic disorders and the use of small molecules to control gene expression . Research will focus on two monogenic diseases , fragile X syndrome and facioscapulohumeral muscular dystrophy , both of which respond to an increase or decrease in proteins . The cancer cells will be used to mimic the cellular processes that have malfunctioned​ in these diseases with altered gene expression and small molecules used to modulate it . Fulcrum CEO Robert Gould said the $ 55 m was needed to advance the platform and get into the clinic .
Third Rock Ventures is launching start-up Fulcrum Therapeutics with funding of $55m to study genetic disorders and the use of small molecules to control gene expression. Research will focus on two monogenic diseases, fragile X syndrome and facioscapulohumeral muscular dystrophy, both of which respond to an increase or decrease in proteins. Skin stem cells will be used to mimic the cellular processes that have malfunctioned​ in these diseases with altered gene expression and small molecules used to modulate it. Fulcrum CEO Robert Gould said the $55m was needed to advance the platform and get into the clinic.