CRISPR Therapeutics Stock Is Up Only 8% This Year. Is Cathie Wood's Bet on the Gene-Editing Stock Dead Money?

CRISPR Therapeutics (NASDAQ: CRSP) is up only slightly more than 8% so far this year, well under the 27% return of the Nasdaq Biotechnology index and slightly below the 12% return of the S&P 500. That's not particularly surprising, since the biotechnology company has one commercialized therapy and has never shown a profit.

Cathie Wood, through her Ark Invest ETFs, is a big backer of CRISPR Therapeutics and owns 94.6 million shares of the stock, at an average price of $66. At today's current price of around $52, she's lost more than a bit on the move. Wood is thinking long-term, however.

The biotech, named for the breakthrough CRISPR gene-editing technique, presents a strong long-term case for investment. Its lead therapy, Casgevy, which treats the rare blood disorders sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TBT), is seeing strong revenue growth. The company has a candidate called CTX611 that's about to enter a phase 3 trial, and nine other therapies in its pipeline, so it could soon have other revenue drivers.

Here are three reasons why Wood's bet on CRISPR Therapeutics isn't dead money.

Image source: Getty Images.

It has a cell-therapy pipeline with strong potential

This past May, CRISPR Therapeutics paid Sirius Therapeutics $95 million for the rights to CTX611. It's a long-acting small interfering RNA (siRNA) therapy that's being tested to prevent thrombosis and thromboembolic disorders.

There is a large population of patients who are at risk for potentially life-threatening thromboembolic events due to underlying co-morbid conditions such as cancer, cardiovascular disease, or hypercoagulability. In the U.S. alone, according to the Centers for Disease Control, there are up to 900,000 people affected by venous thromboembolism (VTE, a blood clot) each year, and roughly 60,000 to 100,000 in the U.S. die each year from VTEs.

That target group is much larger than the one which Casgevy treats. CTX611 silences messenger RNA for coagulation factor XI in the liver, reducing the risk of blood clots by lowering the factor's production. It could avoid side effects such as excessive bleeding that are associated with traditional anticoagulants such as warfarin. Anticoagulants represent a $20 billion market in the U.S.

Another therapy with a relatively broad patient population is CTX310, an in vivo gene-editing treatment that targets the ANGPTL3 gene to lower blood lipid levels for patients with severe hypercholesterolemia and cardiovascular risk.

Its off-the-shelf CAR-T pipeline is a potential game changer

CRISPR Therapeutics' "off-the-shelf" allogeneic (meaning from a donor) CAR-T pipeline, led by next-generation candidates CTX112 and CTX131, offers distinct operational, clinical, and strategic advantages over traditional autologous (from a patient) cell therapies.

CTX112 targets CD19-positive B-cell cancers. It is also being evaluated for severe autoimmune conditions, including systemic lupus erythematosus (SLE) and lupus nephritis, with the aim of resetting the immune system via B-cell depletion.

Off-the-shelf therapies eliminate the weeks-long cell-harvesting, manufacturing, and vein-to-vein delays involved in autologous treatments. Patients with rapidly progressing cancers or severe autoimmune flare-ups can be treated immediately.

Sourcing T-cells from healthy, vetted donors enables a single manufacturing batch to yield dozens of standardized doses. This significantly reduces per-dose production costs, simplifies logistics, and improves scalability. Unlike autologous therapies, which are constrained by a single patient harvest, allogeneic batches allow physicians to redose patients as needed to achieve or maintain remission.

Its finances are improving, albeit slowly

In the second quarter, CRISPR Therapeutics reported revenue of $10.2 million, up from $892,000 in the same period a year ago. It reduced expenses, resulting in a loss per share of $0.94, compared with a loss of $2.40 per share in the second quarter of 2025.

Following a stock sale it's also in a stronger cash position, with $2.36 billion in cash, up 19.2% from the same period a year ago. Through disciplined operational and financial management, the company should be able to fund its broad pipeline through multiple phase 1 and phase 2 clinical readouts without needing additional dilutive equity financing in the near term.

Its main source of revenue is Casgevy profits, shared with its development partner, Vertex Pharmaceuticals, which handles primary commercialization and gives 40% of net profits to CRISPR Therapeutics.

The uptake of Casgevy among patients is slow because the treatment, from cell collection to reinfusion, takes several months. However, the ramp-up in patient initiations and insurance coverage approvals provides visible revenue momentum over multiple quarters.

On July 1, the U.S. Food and Drug Administration (FDA) approved Casgevy for children 2 years of age and older with SCD or TDT -- the first genetic therapy indicated for children as young as 2 for both conditions. With this approval, CRISPR says that 5,500 patients with these disorders may be eligible for treatment with Casgevy for the first time.

Near-term expansion into pediatric patient populations would further expand the total addressable market (TAM) for the therapy.

James Halley has positions in CRISPR Therapeutics. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy.