Benefits of gene-editing therapy OTQ923 seen in small Phase 1 trial
Treatment with the investigational gene-editing therapy OTQ923 led to the sustained production of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three adults with severe sickle cell disease (SCD), according to data from a Phase 1 study. The treatment involved collecting red blood cell precursors, called hematopoietic stem…