Vanessa Pataia,  —

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Phase 1/2 Trial of Gene-editing Therapy for SCD Cleared by FDA

The U.S. Food and Drug Administration (FDA) has cleared the start of a Phase 1/2 clinical trial testing a genome editing-based therapy, known as OTQ923, in adults with severe complications of sickle cell disease (SCD). OTQ923, developed by Novartis and Intellia Therapeutics, uses the CRISPR/Cas9 genome-editing technology.