Patients Finally Receive Vertex-CRISPR and Bluebird Sickle Cell Gene Therapies

Vertex Pharmaceuticals recently announced that patients have started receiving its gene therapy Casgevy for sickle cell disease, marking a significant development in the field. This comes after the company had previously indicated, as of their second-quarter earnings on August 1, that no patients had yet undergone the infusion process. The confirmation from Vertex was reported by BioSpace.

Simultaneously, bluebird bio is also administering its FDA-approved sickle cell gene therapy, Lyfgenia, to patients. The New York Times reported that patients are beginning to receive their edited cells, which signifies another milestone for the treatment of sickle cell disease. Both therapies were approved on the same day, creating a competitive landscape for gene therapy in this medical area.

These advancements are seen as breakthroughs for sickle cell treatment, although experts have anticipated that the adoption of these gene therapies would be slow due to the lengthy and complex process, which can take as long as a year. Stuart Arbuckle, Vertex's Chief Operating Officer, noted during an earnings call that the journey from patient interest to the actual infusion of edited cells is intricate and prolonged. Despite this, there is a high level of interest from patients, physicians, and other stakeholders.

Vertex has not disclosed the number of patients who have received Casgevy. However, during their Q2 earnings call, Vertex revealed that they had activated 35 treatment centers globally, with a target of 75 centers. Approximately 20 patients had their cells collected, indicating they are progressing towards receiving the therapy. The patients are from various regions, including the U.S., Europe, and the Middle East. Arbuckle also mentioned efforts to enhance reimbursement processes to ensure patient coverage across different jurisdictions.

The timeline for when Casgevy and Lyfgenia will start generating revenue is being closely monitored. Vertex aims to transform its early patient interest into substantial sales, with William Blair estimating peak sales for Casgevy to reach $3.6 billion, notwithstanding the slow initial uptake. Arbuckle expressed confidence that Casgevy would significantly benefit a large number of patients globally, presenting a multibillion-dollar opportunity.

On bluebird bio's side, the company announced during their second-quarter earnings call on August 14 that four patients had completed cell collection, the first step toward receiving Lyfgenia. The company expects that it will take about two quarters from the initial cell collection to recognize revenue. James Sterling, bluebird bio’s CFO, stated that interest in their therapies is accelerating, and they have a clear path to converting patient starts into a consistent revenue stream. He emphasized that the slower-than-expected rollout is not due to lack of demand but rather the time required for patients to schedule their treatments around significant life events, given the commitment involved in undergoing gene therapy.

Overall, the initiation of these gene therapies represents a crucial step forward in treating sickle cell disease, with both companies working diligently to overcome the challenges associated with the rollout and to ensure that the treatments reach patients efficiently and effectively.