As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their CRISPR-based gene-editing therapy Casgevy, one doctor who treats sickle cell disease patients is already seeing strong demand. As the rollout gains steam, that demand could create a supply bottleneck, he figures.
Speaking on a recent webinar hosted by Evercore ISI analysts, Anu Agrawal, M.D.—a hematologist and oncologist at the University of California San Francisco—said he’s already accumulated a waiting list of 15 to 20 patients at his site. As the launch progresses, the doctor believes demand will initially outpace supply, the analysts wrote in a Wednesday note summarizing the discussion.
Agrawal’s center cares for hundreds of SCD patients and is currently a "center of excellence" for bluebird bio’s beta thalassemia gene therapy Zynteglo. The center hopes to soon score the same reputation around Casgevy, the analysts wrote.
The doctor's ambition is to primarily focus on younger patients, where Vertex and CRISPR’s gene therapy could have the “biggest impact,” according to the analyst note.
When it comes to early hurdles for the drug’s launch, Agrawal highlighted public-payer budget concerns, plus manufacturing and hospital staffing.
At his own practice, Agrawal hopes to treat 1 or 2 SCD patients with Casgevy over the first year of the drug’s launch, then up to 16 patients per year across multiple gene therapies.
Vertex and CRISPR did not immediately respond to Fierce Pharma’s request for comment on the doctor's predictions.
Meanwhile, the companies have kept busy with their efforts to support the groundbreaking launch.
In one manufacturing partnership, the Casgevy team tapped Charles River Laboratories to help make the new medicine. Earlier this week, the CDMO said it had snagged regulatory clearance to crank out Casgevy from its Memphis production plant.
Prior to that, Vertex enlisted Scottish stem cell expert RoslinCT to tackle commercial production duties on its gene-edited therapy. RoslinCT, which is the CDMO arm of the famed Roslin Institute, boasts two manufacturing facilities—one in Edinburgh, Scotland, and the other on the outskirts of Boston.
Producing Casgevy is far from easy, with each batch uniquely tailored to the individual patient. But the therapy is intended to be a one-time treatment, replacing the cumbersome process of therapy by way of blood transfusions.