sickle cell (52)

FDA Accepts Investigational New Drug Application for CRISPR/Cas9-Based Sickle Cell Disease Therapeutic Candidate Developed Under Collaboration with Intellia Therapeutics

Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application submitted by its collaborator, Novartis, for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease (SCD). This Phase 1/2 clinical trial will begin investigating OTQ923 in adult patients with...