CRISPR Therapeutics' only approved product may experience increased demand.
The biotech could also see significant clinical progress with some key candidates.
Although the stock might jump on positive developments, there is plenty of downside risk as well.
Should you invest in CRISPR Therapeutics (NASDAQ: CRSP) before the end of the year? The company's shares have slightly underperformed the broader market to date, but the biotech may see several catalysts that could send its share price jumping over the next four months. Here are two reasons why investors might want to consider loading up on CRISPR Therapeutics right now.
Unlike most of its similarly sized peers that specialize in gene editing, CRISPR Therapeutics has launched a product, Casgevy, which earned approval in 2023. Casgevy treats two rare blood diseases: transfusion-dependent beta-thalassemia (TDT) and sickle-cell disease (SCD). CRISPR Therapeutics collaborated with Vertex Pharmaceuticals (NASDAQ: VRTX) to develop it. Though the medicine was a breakthrough, it hasn't generated much sales yet. Here are two reasons why.
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First, ex vivo gene-editing therapies are challenging to administer. The process involves collecting patient cells, editing them, and reinserting them into the patient. It takes months to go through all these steps. Casgevy is also expensive. A single treatment course goes for $2.2 million in the U.S. Second, Casgevy's initial indication was fairly narrow. It was approved to treat patients with SCD and TDT aged 12 and older. CRISPR Therapeutics and Vertex just made progress with one of those issues.
Earlier this year, the U.S. Food and Drug Administration expanded Casgevy's label to include patients as young as two. This will allow CRISPR Therapeutics to target patients before TDT and SCD have caused them significant health problems. We shouldn't expect Casgevy's sales to soar later this year. But if CRISPR Therapeutics sees increased demand for the medicine and expands third-party coverage following the recent label expansion, that could meaningfully move the stock price.
It's critical to keep an eye on Casgevy's progress, but CRISPR Therapeutics clinical work will be an even more important catalyst. The company should release data from several ongoing clinical trials by year-end. For instance, CRISPR Therapeutics is developing CTX611, a potential anticoagulant. CTX611 is one of the few candidates in CRISPR Therapeutics' portfolio that isn't a gene editing product.
CTX611 is an RNA-silencing medicine. Therapies of this kind work by "switching off" the instructions for making certain proteins involved in some conditions. In this case, CTX611 targets the Factor XI protein involved in blood clotting. Here's why it could be a breakthrough.
Whereas most anticoagulants are taken daily and carry a risk of severe bleeding, CTX611 is being developed to be administered twice a year and could also reduce the main side effects of its competitors. CRISPR Therapeutics is currently testing CTX611 in phase 2 clinical trials in patients who underwent total knee arthroplasty, a procedure that can lead to blood clots.
The company plans to release data from this study before the end of the year. Strong results could send CRISPR Therapeutics' share price soaring.
Strong progress with Casgevy and across CRISPR Therapeutics' pipeline could jolt the stock. However, there are significant risks. Clinical setbacks will obviously have the opposite impact. Still, my view is that CRISPR Therapeutics' prospects look somewhat attractive. The company has a reasonably deep pipeline for a biotech company of its size even beyond CTX611. It has already shown it can launch a gene-editing medicine, something that has proven challenging for others despite significant progress in the field.
Over the next few years, we could see CRISPR Therapeutics rack up enough clinical wins to expand its approved portfolio, and even the occasional setback -- which every biotech company experiences -- might not be too damaging, considering its deep pipeline. The stock should appeal to investors with above-average risk tolerance, who should consider initiating a position before the end of the year.
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Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy.