CRISPR Therapeutics Stock Trails Biotech as Casgevy Sales Climb

A gene-editing leader stuck in neutral

CRISPR Therapeutics' stock has lagged the rest of biotech in 2026, even as its business has improved in ways that are easy to measure. By late August the shares were up a little more than 8% for the year. Over the same stretch the Nasdaq Biotechnology index returned about 27% and the S&P 500 returned about 12%2. That gap has prompted the question of whether Cathie Wood's long-running bet on the company has turned into dead money.

Wood's Ark Invest has kept buying. In February Ark added roughly 247,000 shares over two days6. In March it bought about 280,600 shares, worth around $14.8 million, after the stock fell 10% on news of a stock offering3, and another 438,000 shares followed later that month5. By July Ark held about $270 million of CRISPR stock in its flagship Ark Innovation ETF and another $92 million in the Ark Genomic Revolution ETF1. On October 9 the Genomic Revolution fund added 21,487 shares, valued at about $1.08 million8. That works out to roughly $50 a share, about where the stock sat in late summer.

The short answer is that the stock has gone sideways because the market wants proof that CRISPR can build a second business, not because the first one is failing. The next few months bring the data that could supply that proof.

Casgevy is finally selling

Casgevy, the sickle cell and beta thalassemia treatment CRISPR developed with Vertex Pharmaceuticals, was a slow seller for a long time. It brought in about $10 million in 2024, its first full year on the market, and $115.8 million in 202516. This year the growth is real. Casgevy generated $43 million in the first quarter, and more than 500 patients worldwide had started the treatment process by then15. Second-quarter revenue rose to $76 million, up 78% from the previous quarter and 151% from a year earlier20.

The biggest recent boost was a label expansion. The FDA approved Casgevy for children as young as 2 just 53 days after the filing, which the companies say makes about 5,500 more patients eligible20. The therapy is now approved in 39 countries, and Vertex has won reimbursement in Germany for patients 12 and older11.

Only part of that money reaches CRISPR. Vertex makes and sells Casgevy, and the two companies split profits and program costs 60/40, with Vertex taking the larger share15. That is why CRISPR's own reported revenue looks small. Total revenue was $10.2 million in the second quarter, compared with $892,000 a year earlier20. The company's share of Casgevy shows up instead as a lower collaboration expense, which fell to $40.3 million from $45.2 million20.

Some coverage highlighted a roughly 1,000% jump in revenue2. That figure measures milestone and grant payments, not the commercial business, so investors are right not to treat it as a growth rate.

The actual numbers are still encouraging. The net loss for the second quarter shrank to $91.2 million from $208.5 million a year earlier11. Cash and securities stood at about $2.36 billion at the end of June20. Casgevy's first-half sales came to about $119 million. William Blair's model, issued early in the year, calls for $344 million in Casgevy revenue for all of 2026, with $132 million going to CRISPR16. Hitting that would require a much stronger second half. Vertex executives have warned that quarterly results will swing because revenue is only booked when a patient is infused, sometimes months after starting treatment. They expect the pattern to smooth out in 202716.

Why the stock hasn't followed

The disconnect has fairly ordinary causes. CRISPR has one commercial product, has never made a profit2, and has been raising money. A stock offering in March knocked the shares down 10%3, and the company also sold $600 million of convertible notes due in 2031, convertible at $76.56 a share5. The note sale brought in about $585 million in net proceeds and pushed cash to $2.44 billion at the end of March17. That cushion was welcome, but it came at the cost of dilution and a share-conversion price that now acts as a reference point well above the current stock price.

Analysts are divided. Barchart counted 29 analysts in March: 15 rated the stock a Strong Buy, 12 a Hold, and one a Moderate Sell5. Piper Sandler raised its target to $1105. Bernstein stayed at Market Perform with a $56 target7. Morgan Stanley's June upgrade from Underweight to Equal Weight, with its target raised from $33 to $60, read more like a skeptic coming around than a call to buy. The firm said it wanted clearer visibility on the Casgevy launch and pointed to CRISPR's in vivo programs, which edit genes directly inside the body, as the area to watch7.

Within the year, the stock fell about 11% by mid-March5 and traded between $44.12 and $78.48 over the past 52 weeks1. The roughly 8% gain counted in August was not a steady climb. It was a recovery from a drawdown.

Coverage of the pipeline doesn't always match the company

The bullish coverage gets some details wrong. One widely shared August analysis said CTX611, an RNA-based drug designed to lower clotting factor XI, was "about to enter a phase 3 trial"2. CRISPR's own guidance says top-line Phase 2 results in knee-replacement patients are expected in the second half of 202627. That leaves late-stage testing at least one readout away. The same piece filed CTX611 under the cell-therapy pipeline2, although it is a small interfering RNA (siRNA) drug developed with Sirius Therapeutics24. It also said Ark owns 94.6 million shares at an average cost of $662. That would be close to CRISPR's entire share count, which averaged about 96 million in the first quarter17. It is also hard to square with Ark's reported holdings of about $362 million a month earlier1. Investors should treat that figure with caution.

A July article described CTX110 as the preclinical successor to zugocabtagene geleucel, the off-the-shelf CAR-T therapy formerly called CTX112 that is now in Phase 1 for lymphoma and autoimmune diseases1. The pros and cons of donor-derived CAR-T are well documented. It avoids the weeks of manufacturing needed for patient-specific therapy and can be produced in batches2, but it competes with approved products from Novartis, Bristol Myers Squibb, and Gilead, and approval is years away1. Where outlets disagree on these details, the company's own disclosures should be the reference point.

CTX310 is the next big test

The in vivo cardiovascular program, led by CTX310, is where the investment case will be decided. CTX310 uses lipid nanoparticles to carry CRISPR components to the liver, where they permanently switch off the ANGPTL3 gene25. Phase 1 results presented at the American Heart Association meeting in November 2025, and published in The New England Journal of Medicine, showed mean reductions of 73% in ANGPTL3, 55% in triglycerides, and 49% in LDL cholesterol at the highest dose28. One-year follow-up presented at the European Society of Cardiology (ESC) Congress on August 28 showed the effect holding. At the top dose, ANGPTL3 was down 79%, triglycerides 48%, and LDL 53%, with no treatment-related serious adverse events among 15 participants30. The main safety signal so far was a temporary rise in a liver enzyme in one patient, which returned to baseline within two weeks25.

The next step is close. Phase 1b data on CTX310 were selected for a late-breaking oral presentation at the American Heart Association meeting on November 929. That trial focuses on severe hypertriglyceridemia and refractory hypercholesterolemia27. The pipeline behind it is growing too. CTX340 for hard-to-treat high blood pressure and CTX460 for alpha-1 antitrypsin deficiency entered Phase 1 in the second quarter20. CTX321, the lipoprotein(a) program, uses a new guide RNA the company says is about twice as potent as the one in its predecessor, CTX32025.

The upside is large if the program keeps working. The company says more than 40 million Americans have elevated LDL, severely elevated triglycerides, or both21. The most bullish analyst models assume revenue of about $5.6 billion by 2029 if the cardiovascular programs scale29. That is speculative for a drug that has so far been tested in 15 people.

Is it dead money?

No, but the stock is waiting on catalysts, and the label fits for now. The case for patience is stronger than the share price suggests. Casgevy is growing quickly, losses are shrinking, and roughly $2.4 billion in cash gives CRISPR time to run several trials without returning to the market soon29. Ark's continued buying shows Wood is treating weakness as a chance to add shares.

The clearer test is the next two months. A strong CTX310 presentation on November 9 and solid Phase 2 data for CTX611 would back the claim that CRISPR is more than a single-product sickle cell company2729. Disappointing results would leave the stock dependent on a Casgevy launch whose quarterly numbers are still uneven16. Investors are not pricing CRISPR as a failure. They are waiting for the in vivo data to show what the platform can do beyond Casgevy.