Casgevy Uptake

Casgevy, the company’s gene-editing therapy for sickle cell disease and transfusion-dependent beta thalassemia, generated $54 million in fourth-quarter revenue and $116 million for the full year.

A total of 64 patients received infusions in 2025, including 30 during the fourth quarter.

Globally, 147 patients initiated treatment through the first cell collection during the year. Patient initiations nearly tripled compared to 2024, reflecting growing momentum heading into 2026.

William Blair noted, “the increase to 147 first cell collections strengthens confidence that higher initiation volumes will translate into materially greater revenue in 2026.”

Pipeline Advancement

Beyond hematology, the company continues to advance its in vivo liver editing programs.

CTX310 remains in Phase 1b trials targeting lipid disorders, while CTX321, a next-generation Lp(a) program, is progressing through enabling studies with updates expected in the second half of 2026.

William Blair analyst Sami Corwin wrote, “We continue to see the company’s in vivo cardiovascular gene editing programs as meaningful value drivers…”

Its siRNA-based candidate CTX611, developed with Sirius Therapeutics, is currently in Phase 2 trials for patients undergoing knee replacement surgery and may have broader applications across thromboembolic diseases.

CRISPR Therapeutics ended 2025 with $1.98 billion in cash and marketable securities. R&D expenses rose to $83.5 million in the fourth quarter, while net loss widened to $130.6 million in the fourth quarter from $37.3 million a year earlier.

CRSP Price Action: CRISPR Therapeutics shares were up 8.81% at $53.24 at the time of publication on Friday, according to Benzinga Pro data.

Photo by T. Schneider via Shutterstock