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.
Early on some had concerns not just about potential hypoxia but also b/c of the aldehyde-based chemistry. I have not heard about this concern lately. Is that something that you have heard come up?
So far I think even with the small N reported on to date the bio-marker data are pretty convincing. Small N is a big issue when evaluating safety though so this is what I worry about most. Pretty devastating disease so would assume AE bar would be relatively high to derail development?
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