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.
It will be a significant moment for sickle cell patients and for Global Blood and its investors. Here are seven things to know about the Global Blood drug, called voxelotor, and the phase 3 clinical trial before results are disclosed:
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