Addressing the slow uptake of gene therapy for sickle cell disease: a lack of interest, a high-cost, or a more complex issue?

INTRODUCTION

The approval of (GT) for sickle cell disease (SCD) marks a major milestone in hematology, offering curative potential without the need for a matched sibling donor. Yet early real-world uptake has been slow. This delay in integration is likely due to complex intertwined barriers that require further elucidation.

AREAS COVERED: We conducted a structured review of the literature, searching PubMed/MEDLINE, Embase, and ClinicalTrials.gov through April 2026 for evidence on the economic, institutional, clinician-level, and patient-level barriers limiting adoption of GT for SCD, as well as global disparities in access.

EXPERT OPINION: Slow uptake of GT reflects the expected challenge of introducing a complex, resource-intensive intervention into a fragmented care system. Addressing these barriers requires coordinated efforts from payers, institutions, clinicians, and patient communities. Without deliberate structural reform, GT risks widening existing health disparities rather than alleviating them.