The Gene‑Therapy Breakthrough Story You'll Be Mad You Ignored at These Prices

Vertex Pharmaceuticals' (NASDAQ: VRTX) stock price is up, but its Casgevy sales partner, CRISPR Therapeutics (NASDAQ: CRSP), is the real buy for long-term investors.

Shares of Vertex, a biotech based in Boston, popped last week after it reported its fourth-quarter results, which included higher 2025 sales for Casgevy, a gene-editing therapy for two genetic blood disorders: sickle cell disease and beta thalassemia. Vertex is already profitable, and makes most of its revenues by selling the only approved drugs for treating cystic fibrosis: Alyftrek, Trikafta, Symdeko, Orkambi, and Kalydeco. The company has also predicted further sales growth for Casgevy this year. Its share price did moderate in the days that followed that report, however, and it gave back most of its short-term gains.

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Switzerland-based CRISPR Therapeutics actually developed Casgevy, the first CRISPR-based gene therapy approved by the Food and Drug Administration. It will also benefit from those increased sales.

Think long-term with CRISPR Therapeutics

There's a reason why Cathie Wood's Ark Innovation ETF (NYSEMKT: ARKK) maintains an approximately 6.3% position in CRISPR Therapeutics; it's the exchange-traded fund's No. 2 holding after Tesla. CRISPR Therapeutics has already shown it has the science to develop groundbreaking gene-editing therapies, and it has five other candidates in clinical trials, all of which have larger potential patient pools than Casgevy.

One of the most promising is its cardiovascular disease therapy candidate CTX310, a single-treatment gene therapy that has been shown to reduce triglycerides and LDL cholesterol by more than 80%. Another gene therapy, currently dubbed CTX320, is being studied as a treatment for atherosclerotic cardiovascular disease.

Anticoagulant SRSD107 is a long-acting gene therapy under development (with collaborator Sirius Therapeutics) to treat patients with thrombosis. And CTX211 is a gene-editing therapy that could restore type 1 diabetes patients' ability to produce insulin.

It should be able to get its candidate therapies over the finish line

CRISPR Therapeutics isn't profitable yet, which helps explain why its shares are down 64% over the past five years. In 2025, it had only $3.5 million in annual revenue, down 91% year over year. However, that was entirely due to an accounting move that the company took in 2024 regarding its collaboration revenue agreements. It also booked a loss of $6.47 per share in 2025, compared to a loss of $4.34 per share in 2024. However, the company has more than $1.9 billion in cash, and if Casgevy sales pick up as expected, it will be able to spend more to develop its pipeline candidates.

The company's therapies have the potential to actually provide functional cures for diseases, not just treat them, which makes their value harder to peg. Casgevy, for example, has a list price of $2.2 million per treatment. CRISPR Therapeutics' progress with Casgevy gives it a leg up on rival biotechs because it already has the manufacturing and treatment infrastructure for gene-editing therapies.

The stock is a high-risk, high-potential-reward investment that will likely remain volatile until the company starts showing a profit, but the company's pipeline also makes it an attractive acquisition target. At its current low price, given its strong upside potential, CRISPR Therapeutics is worth buying.

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