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Global Blood Therapeutics (GBT) is taking an unprecedented regulatory gamble to win approval for a new drug to treat sickle cell disease. Are investors underestimating the risk that this effort blows up?

It looks that way. The South San Francisco-based biotech disclosed mixed results from a Phase 3 clinical trial in late June. Since then, Global Blood executives have been on the road with investors, talking up an FDA submission strategy for its drug, called voxelotor, that has never been tried before in sickle cell disease.

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