FDA Approves First-Ever CRISPR Gene-Editing Treatment for Sickle Cell Disease

The groundbreaking treatments, Casgevy and Lyfgenia, offer hope for a cure but come with a hefty price tag, raising concerns about accessibility.

The U.S. Food and Drug Administration (FDA) has approved the first-ever CRISPR gene-editing treatment for sickle cell disease (SCD), a painful and potentially fatal blood disorder that affects mostly Black Americans.
The gene-editing treatment, called Exa-cel and marketed as Casgevy, is developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It involves taking stem cells, cultivating them in a lab, using a form of molecular scissors to slice the offending DNA regions that lead to the malformed hemoglobin at the root of SCD, and then re-infusing them into a patient over the course of a month with continuous medical attention.
Alongside Casgevy, the FDA also approved Bluebird Bio’s lovo-cel (marketed as Lyfgenia), which uses a different type of technology to replace the damaged gene that causes sickle cells to form.
Both treatments have produced similar, and impressive, clinical trial results with few safety concerns. For Casgevy, regulators cited data showing that 29 of 30 patients had achieved the study’s main goal, which was to see patients free from those painful crisis events requiring hospitalization for at least one year.
The cost for the treatments is a critical question, with list prices averaging anywhere from $1 million to $2 million. Vertex has listed the wholesale price of Casgevy as $2.2 million while Bluebird said in a statement that Lyfgenia’s wholesale price will be $3.1 million.