Two gene therapies for sickle cell disease (SCD) have submitted biologics license applications, marking a significant advance in treating this debilitating condition. SCD, characterized by pain and a shortened lifespan, affects an estimated 100,000 individuals in the United States, primarily Black patients. Despite the availability of disease-modifying treatments (DMTs) like hydroxyurea and recent additions such as Endari and Oxbryta, only 31% of severe SCD patients receive DMTs, highlighting the underserved nature of this community. The lack of research funding, healthcare provider competency, and comprehensive care centers contribute to this disparity.

The potential game-changer lies in one-time gene therapies, Lovo-cel and exa-cel, currently under FDA review. These therapies have the potential to free patients from lifelong treatment by addressing the root genetic causes of SCD. While DMTs have improved quality of life, gene therapies offer the promise of curing the disease, albeit with certain drawbacks, including myeloablative conditioning. Despite these challenges, the long-term durability and affordability of these gene therapies hold promise for improving the lives of SCD patients.

Read more: https://www.managedhealthcareexecutive.com/view/sickle-cell-disease-finally-getting-its-due

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