CRISPR Therapeutics

Casgevy is the first ever approved medicine built on CRISPR gene editing, and the results are remarkable. In the long term studies, 100 percent of sickle cell patients stayed free of painful crises for at least a year, and more than 98 percent of beta thalassemia patients no longer needed blood transfusions. Regulators just expanded approval to children as young as two, widening the pool of patients who can be treated.

This is a biotech that is not about to run out of cash. The company holds about 2.4 billion dollars in cash and investments after a recent fundraise, enough to fund roughly five years of research at the current spending pace. That cushion lets it push a deep pipeline of experimental treatments for heart disease, cancer, and autoimmune conditions without being forced to sell stock at a bad time.

CRISPR is not profitable and loses a lot of money. It lost about 123 million dollars in the first quarter of 2026 alone, because its share of Casgevy sales is still small while research costs stay high. Casgevy revenue was only about 43 million dollars for the quarter, and that is actually down from the quarter before, so the road to real profits is going to take years.

The treatment itself is a tough journey for patients. It costs about 2.2 million dollars, takes up to a year to complete, and requires chemotherapy that can cause infertility, so uptake has been slow. Only about 500 people worldwide have started treatment so far, and rivals like Beam Therapeutics are racing to build gentler versions that could take market share.