Vertex, CRISPR’s Casgevy Highlights Complex Path to Gene Therapy Profitability
By Annalee Armstrong,
BioSpace
| 09. 11. 2024
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...
Related Articles
By Jonathan Matthews, GMWatch | 12.11.2025
In our first article in this series, we investigated the dark PR tactics that have accompanied Colossal Bioscience’s de-extinction disinformation campaign, in which transgenic cloned grey wolves have been showcased to the world as resurrected dire wolves – a...
By Jenny Lange, BioNews | 12.01.2025
A UK toddler with a rare genetic condition was the first person to receive a new gene therapy that appears to halt disease progression.
Oliver, now three years old, has Hunter syndrome, an inherited genetic disorder that leads to physical...
By Simar Bajaj, The New York Times | 11.27.2025
A common cold was enough to kill Cora Oakley.
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare genetic condition that leaves the body without key white blood cells.
It’s better...
By Rachel Hall, The Guardian | 11.30.2025
Couples are needlessly going through IVF because male infertility is under-researched, with the NHS too often failing to diagnose treatable causes, leading experts have said.
Poor understanding among GPs and a lack of specialists and NHS testing means male infertility...
