Gene therapy for henoglobinopathes, a middle-income country perspective.

Access to gene therapy for hemoglobinopathies—such as sickle cell disease and thalassemia—offers transformative potential for patients in middle-income regions, where disease burden is high but access to advanced care remains uneven. Recent scientific breakthroughs have moved gene therapy from experimental trials to approved treatments; however, access is severely constrained by cost, infrastructure requirements, and regulatory capacity. Cost remains the dominant barrier, with prices reaching several million dollars per patient in Europe and North America. These prices are widely viewed as reflecting profit expectations in high-income biotech markets rather than the true cost of delivering the procedure.

Encouragingly, alternative models are emerging. Chinese companies are reportedly offering gene therapy for under USD 500,000, while research centers in India are developing approaches that could reduce costs below USD 100,000. Although still unaffordable for out-of-pocket payment, such prices may become viable under universal health coverage, where costs are distributed across large risk pools. In India, however, even standard bone marrow transplantation—available for USD 10,000–15,000—is accessed by only about 5% of eligible low-risk patients, highlighting the long road ahead. Strategic partnerships among governments, global health organizations, industry, and patient advocates will be essential to ensure gene therapy narrows, rather than widens, global health inequities.