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BCL11A-directed gene therapy advances in sickle cell disease

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.


Esrick EB et al. ASH 2019. Abstract LBA-5.