Following FDA approval, Vertex Pharmaceuticals and CRISPR Therapeutics, along with Bluebird Bio, have initiated infusions of their respective gene therapies for sickle cell disease. This marks a significant step forward in the treatment of this debilitating condition, offering new hope to patients who have long awaited more effective options.
First Patients Receive Gene Therapies
Vertex confirmed that patients have begun receiving Casgevy, their CRISPR-based gene therapy, after initial concerns about delays following its approval. Bluebird Bio also reported that patients are now receiving infusions of Lyfgenia. These developments signal a turning point in the treatment landscape for sickle cell disease.
Complexities of Treatment Uptake
Experts anticipated a slow uptake of gene therapies due to the lengthy and complex treatment process, which can take up to a year. Stuart Arbuckle, Chief Operating Officer at Vertex, acknowledged the complexity, stating, "We have always known that Casgevy offers an enormous advancement for patients... the patient journey... is long and complex."
Market Expectations and Revenue Projections
Despite the slow start, analysts are optimistic about the financial prospects of both therapies. William Blair estimates that peak sales for Casgevy could reach $3.6 billion. Bluebird Bio CFO James Sterling indicated that investors can expect approximately two quarters between initial cell collection and revenue recognition for Lyfgenia.
Patient Demand and Treatment Timelines
Sterling clarified that the pace of treatment initiation is not due to a lack of demand but rather the time-consuming nature of the process. Patients are scheduling treatments months in advance, often aligning with significant life events, given the commitment required for gene therapy.
