Revolutionizing Sickle Cell Disease Treatment: Unveiling CRISPR and Lentiviral Therapies—Navigating Complexities in Access, Equity, and Global Health Dynamics

Abstract

Advancing Sickle Cell Disease Treatment: Addressing Access Challenges In this editorial, we discuss the recent approval by the U.S. Food and Drug Administration (FDA) of two new treatments, Casgevy and Lyfgenia, for sickle cell disease (SCD) in patients aged 12 and older. These treatments are significant because Casgevy is the first FDA-approved therapy to use a new genome editing technology. This approval represents a big step forward in gene therapy and in the treatment of sickle cell disease which is clinically characterised by the complications of an unresponsive hemoglobin. However, while celebrating this achievement, we need to think about how to make sure everyone who needs these treatments can get them, especially in Africa where SCD affects many people. It's not just about making the treatments, but also about making sure they reach the people who need them the most. This means we need to work on things like logistics (how to get the treatments to the right places) and political will (making sure governments support getting these treatments to everyone who needs them). In the end, it's not enough to have these new treatments available - we need to make sure they actually help the people who need them most, wherever they are in the world.