Vertex, CRISPR’s Casgevy Highlights Complex Path to Gene Therapy Profitability

Complex gene therapies are starting to hit the market but all have faced the same reality: a tepid reception from the healthcare system and a cloudy path to profitability.

It can take about a year for a patient to go through the preparations needed to receive a gene therapy treatment, Jen Klarer, managing partner at The Dedham Group, told BioSpace. “I expected there to be a slow time from approval to treating the first patient.”

CRISPR Therapeutics and Vertex Pharmaceuticals are a great example. The companies made history last year when they achieved the first approval of a CRISPR-Cas9 gene editing therapy for sickle cell disease in December 2023. But when the companies reported 2024 second quarter earnings a few months later, the sobering—albeit expected—reality set in: no patients had been treated.

The fuller picture of Casgevy’s path to profitability is even more complicated. During Vertex’s second quarter earnings call, the company explained that 20 patients are “in the funnel” to receive Casgevy. That means that the patients have signed up and gone through the cell collection process. This...