Key clinical point: Preliminary results of a small pilot study show that a gene therapy approach that targets a major repressor of fetal hemoglobin is acceptably safe and appears to mitigate the pathology of sickle cell disease.

Major finding: Following gene therapy, treated patients have had no instances of vaso-occlusive pain crises, respiratory events, or neurologic events. No patients have required transfusion, except one with severe underlying vascular disease for whom post–gene therapy transfusions were planned.

Study details: A single center pilot/feasibility study involving five patients with sickle cell disease.

Disclosures: Dr. Esrick reported having no financial disclosures. Her coauthors reported disclosures related to Alerion Biosciences, Novartis, Orchard Therapeutics, Roche, AstraZeneca, and bluebird bio, among others.