Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

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Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a Phase 1/2 clinical trial. The U.S. Food and Drug Administration (FDA) has cleared GPH101 for clinical testing. With this decision, the therapy’s developer, Graphite Bio, has now become the first company to advance to trial a lead investigational treatment that would address the disease’s underlying cause. The new, open-label trial, called CEDAR, is…

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