For the estimated 100,000 Americans living with sickle cell disease, June's awareness month arrives with news that would have seemed like science fiction just a decade ago: a functional cure now exists.
Sickle cell disease is a hereditary blood disorder in which red blood cells take on an abnormal crescent shape, clumping together and blocking blood flow. The result is episodes of severe pain, organ damage, stroke, and a significantly shortened lifespan. Until recently, the only cure was a bone marrow transplant, a risky procedure that requires a matched donor and is unavailable to most patients.
That changed in late 2023 when the FDA approved two landmark gene therapies: Casgevy, developed by Vertex Pharmaceuticals, and Lyfgenia from bluebird bio. Casgevy made history as the first treatment ever approved using CRISPR gene-editing technology, the molecular "scissors" that can precisely rewrite a patient's own DNA. Both therapies work by collecting a patient's stem cells, genetically correcting them in a laboratory, and reinfusing them. The repaired cells then produce healthy red blood cells.
Clinical results have been striking. In trials, the majority of patients treated with Casgevy experienced no severe pain crises for at least a year after treatment. Vertex recently announced the therapy is showing equally promising results in children as young as five.
Now for the gasp: Casgevy carries a list price of $2.2 million for a single course of treatment. Lyfgenia comes in at $3.1 million.
The federal government is working on it. In 2025, CMS launched a program in 33 states to make gene therapy accessible through Medicaid via outcomes-based payment agreements. Progress, but for now, the cure exists in a place not everyone can reach.
