FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

Joana Carvalhoby Joana Carvalho

In Sickle Cell Disease News.

FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

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FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigating EDIT-301, Editas Medicine’s experimental gene editing cell therapy for  sickle cell disease (SCD). The planned open-label study, to be called RUBY, will assess the safety and efficacy of a single dose of EDIT-301 in patients with severe SCD. “The FDA’s clearance for initiation for our EDIT-301 clinical trial is an exciting moment for us and the patients we hope…

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