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While genome editing is having a moment, particularly with reports at the beginning of this year of success in the first clinical trials for sickle cell disease (SCD), it’s worth remembering that this is not the only front on which progress is being made in treating this inherited blood disorder.
In 2019, the San Francisco biotech company Global Blood Therapeutics (GBT) won approval for its small-molecule drug Oxbryta, developed in near-record time. Oxbryta is not so much treating the pain crises experienced by SCD patients as the underlying polymerization of the sickle globin subunits.
The CEO of GBT is Ted Love, MD, who grew up in rural Alabama. He enjoyed a successful career at several biotech companies including Onyx, Nuvelo, and Genentech, before coming out of semi-retirement to take the reins of GBT as president and CEO.
In “Close to the Edge” (Episode 4), Love spent an hour talking about GBT’s mission and the sad history of disdain and discrimination faced by SCD patients, most of whom are of African descent. This ranges from patients seeking help from health professionals while in pain crises to the chronic underfunding of SCD research compared to other genetic diseases such as cystic fibrosis.
Love staunchly defended Oxbryta’s list price of $125,000/year, which has sparked some criticism, as GBT continues its research on additional therapies. Work on other drug candidates is progressing well, while GBT has also in-licensed a couple of promising programs from Sanofi’s Bioverativ subsidiary.
More than 70 years after SCD was described by Nobel Laureate Linus Pauling as the first molecular disease, Love is leading the charge to make a difference.