Virtual Clinical Trials Harvard is launching start-up Fulcrum Therapeutics with funding of $ $55m m to study genetic disorders and the use of small molecules to control gene expression. Research will focus on more than a third 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 Gould said the 55 was needed to advance the platform and get into the clinic.
Harvard is launching start-up Fulcrum Therapeutics with funding of $ $55m m to study genetic disorders and the use of small molecules to control gene expression. Research will focus on more than a third 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 Gould said the 55 was needed to advance the platform and get into the clinic.