In a major milestone for sickle cell disease treatment, patients are finally receiving infusions of two groundbreaking gene therapies: Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, and bluebird bio’s Lyfgenia. This marks a crucial step forward after both therapies received FDA approval on the same day.
Vertex-CRISPR’s Casgevy: Infusions Underway
As of the second-quarter earnings call on August 1, Vertex had reported that no patients had yet received Casgevy, a gene therapy designed to tackle the genetic cause of sickle cell disease. However, Vertex has since confirmed that the first patients have now been treated, signifying the therapy’s official launch into clinical practice.
Casgevy works by using CRISPR gene-editing technology to modify the patient’s own cells, aiming to eliminate the sickle-shaped red blood cells that cause painful blockages in blood vessels. Vertex’s Chief Operating Officer, Stuart Arbuckle, acknowledged that while the patient journey is long and complex, the treatment represents an enormous advancement for sickle cell patients.
“We have always known that Casgevy offers an enormous advancement for patients,” Arbuckle said during the earnings call. “We’ve also consistently communicated that the process from patient interest to infusion of edited cells is long and complex. Whilst it’s still early in the launch, we have gained many learnings, and interest is high among patients, physicians, and governments.”
While Vertex did not provide specific numbers on how many patients have received Casgevy to date, they have noted that 35 treatment centers have been activated globally, with a goal of increasing that number to 75. As of the latest updates, approximately 20 patients have had their cells collected, indicating they are in the early stages of treatment.
bluebird bio’s Lyfgenia: A Head Start?
bluebird bio, which is also rolling out a sickle cell gene therapy—Lyfgenia—is seeing similar progress. The company had the advantage of established treatment centers from its two other FDA-approved gene therapies, which has facilitated the faster setup of Lyfgenia treatment centers.
As of bluebird’s second-quarter earnings call on August 14, four patients had already undergone cell collection, a crucial first step in their journey toward receiving Lyfgenia. A report from The New York Times also confirmed that patients are now beginning to receive infusions of their own edited cells.
Similar to Casgevy, Lyfgenia involves collecting the patient’s own cells, editing them to correct the genetic defect that causes sickle cell disease, and reintroducing these corrected cells into the patient’s body.
Revenue Outlook for Casgevy and Lyfgenia
Both companies are under scrutiny from investors eager to understand when the therapies will start generating significant revenue. For Vertex, analysts estimate peak sales of Casgevy could reach $3.6 billion, despite the slower-than-expected start. Arbuckle assured investors during the earnings call that Vertex has been working diligently to improve reimbursement processes in all regions, including the U.S., Europe, and the Middle East, to ensure patients can access the therapy.
At the time of the call, Vertex had approximately 20 patients in the treatment funnel, with ongoing efforts to expedite the process. Investors can expect a gradual ramp-up as treatment centers continue to activate and reimbursement pathways are solidified.
Similarly, bluebird bio’s CFO James Sterling emphasized that Lyfgenia’s slower start is more of a timing issue rather than a lack of demand. “Patients are scheduling their treatment many months in advance, often around life events given the time commitment involved in undergoing gene therapy,” Sterling said. bluebird bio expects about two quarters between initial cell collection and revenue recognition.
Both therapies represent a breakthrough in the treatment of sickle cell disease, and their gradual rollout reflects the complexity of delivering such advanced medical interventions. While the process may take time, the strong patient interest and initial progress signal a transformative future for sickle cell patients worldwide.
These gene therapies are not just a scientific triumph; they offer hope for a potential cure to a disease that has long been characterized by chronic pain and limited treatment options. As more patients begin to receive these innovative therapies, the impact on the sickle cell community will likely be profound.
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