Patients Finally Start Receiving Vertex-CRISPR and Bluebird Sickle Cell Gene Therapy

As of August 1, no patients have received an infusion of Vertex Pharmaceuticals and CRISPR Therapeutics’ sickle cell gene therapy Casgeva, according to second-quarter results. However, infusions have already started, a Vertex spokesman confirmed Biospace.

Meanwhile, at partner company Bluebird Bio, which has received FDA approval for its sickle cell gene therapy Lyfgenia the same day as CasgevyPatients are also starting to receive infusions of their own edited cells, New York Times reported on Monday.

These moments represent a breakthrough in the treatment of sickle cell anemia. Experts expected an increase in interest gene therapies, which are slow because the process of getting treatment can take up to a year. Vertex Chief Operating Officer Stuart Arbuckle acknowledged this during an earnings conference call last month.

“We’ve always known that Casgevy offers a huge advancement for patients. We’ve also consistently communicated that the patient journey, from patient interest to injection of edited cells, is long and complex. Although it’s still early days, we’ve learned a lot. The level of interest is high among patients, physicians, governments and other stakeholders,” Arbuckle said.

At the time of publication, Vertex had not responded to requests for more information, including how many patients had taken Casgevy.

As for the bluebird that was Faster implementation is expected Lyfgenia, because it already had treatment sites for two other FDA-approved gene therapies, the company announced during its second-quarter financial results conference call on Aug. 14 that four patients had had cells collected as the first step in their Lyfgenia treatment.

When will Casgevy and Lyfgenia start generating revenue?

On its second-quarter earnings call, Vertex activated 35 sites, with a goal of activating 75 worldwide. About 20 patients volunteered their cells for collection, placing them “in the hopper” for treatment. Vertex was not specific, but said the patients came from all regions where the drug is approved, including the United States, Europe and the Middle East. Arbuckle noted on the call that the company was also working to improve reimbursement in all jurisdictions to ensure patients are covered.

“The treatment process is time-consuming, but we are now even more confident that Casgevy will help a large number of patients around the world and represents a multi-billion dollar investment opportunity,” Arbuckle said.

Investors were eager to see when Casgevy’s treatment would begin, and analysts peppered executives with questions on earnings conference calls, trying to get a sliver of information. William Blair estimated that peak sales of the gene therapy could reach $3.6 billion, even with a slow start.

As for Lyfgenia, bluebird bio CFO James Sterling said on the call that investors can expect about two quarters between initial cell collection and revenue recognition. “We’re on track with growing interest in our therapies, and we have a clear path to turn patient launches into a growing, sustainable revenue stream over time.”

Sterling noted that patients are planning their treatments months in advance, often based on life events, taking into account the time commitment for gene therapy. “So it’s not a slower start than expected, and it’s not a demand issue. The demand is actually high. It’s just a matter of time,” he said.