People with sickle cell disease have few effective treatment options. The FDA has only approved two drugs — 20 years apart — to treat the inherited blood disorder. Efforts to develop a cure for sickle cell disease using gene therapy or genome-editing have shown early promise but are still years away from being proven.
A new and novel medicine from Global Blood Therapeutics (GBT), a small biotech based south of San Francisco, could soon fill that sickle cell treatment gap. A late-stage clinical trial is nearing an interim but important readout before the end of the second quarter.