Forget Casgevy: CRISPR Therapeutics' Heart-Disease Data Could Matter More

CRISPR Therapeutics (NASDAQ: CRSP) has a major claim to fame. It developed a medicine called Casgevy, which in 2023 became the first approved therapy to use the Nobel Prize-winning CRISPR gene-editing technique. Casgevy is currently CRISPR Therapeutics' only approved medicine, and it shares the rights to it with Vertex Pharmaceuticals (NASDAQ: VRTX). This therapy is important to CRISPR Therapeutics' prospects, but the company arguably has even more critical catalysts on the horizon.

Notably, the mid-cap biotech has several promising candidates in the pipeline that could make significant progress over the next few years. Let's discuss one of them: CTX310. Here's why this investigational medicine is, perhaps, even more important to CRISPR Therapeutics' future.

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A massive potential addressable market

Casgevy was no doubt a breakthrough, but its commercial opportunity is fairly limited. CRISPR Therapeutics and Vertex Pharmaceuticals have noted that the medicine currently has an addressable market of roughly 60,000 patients across several countries where it is approved, including the U.S., Canada, and several European and Middle Eastern countries.

But CRISPR Therapeutics' CTX310 -- which also utilizes the CRISPR mechanism -- could have a much larger market. This medicine is being designed to decrease LDL cholesterol and triglycerides (TGs), which, at high levels, can cause severe, sometimes life-threatening, cardiovascular and metabolic problems. So, it could reduce the risk of cardiovascular disease in eligible patients. There are approved medications to help patients lower TGs and LDL, most of which are taken once or twice daily.

Nothing wrong with that, but a one-time therapy that addresses this problem once and for all could be a significantly more convenient option than current standards of care. That's what CTX310 could be. What's the addressable opportunity for this medicine? According to CRISPR Therapeutics, more than 40 million patients in the U.S. alone have high LDL or TG levels.

So, this medicine's commercial opportunity could be far larger than Casgevy's for the biotech, especially since CRISPR Therapeutics is developing it alone and won't have to share its profits with another drugmaker, as it does with Casgevy. CTX310 has, so far, posted encouraging results in clinical trials. In August, CRISPR Therapeutics reported data from a Phase 1a clinical trial in patients with high LDL or TG levels associated with certain genetic conditions. The medicine showed a mean TG reduction of 48% and a mean LDL reduction of 53% at the highest dose.

CRISPR Therapeutics will report data from a Phase 1b study in early November. There is still a long way before this therapy can hope to earn approval, but it is important to CRISPR Therapeutics' prospects, so it will be critical to monitor its clinical progress.

Is CRISPR Therapeutics' stock a buy?

CRISPR Therapeutics has underperformed the broader equity market over the past five years. But the company may bounce back in the medium term, provided it can make significant progress in its pipeline. The biotech has other candidates besides CTX310. For instance, it is working on a medicine called CTX611, an investigational, long-acting anticoagulant. Further, over the next five years, CRISPR Therapeutics could also make commercial progress with Casgevy, especially since a recent label expansion in patients as young as two added 5,500 people to its addressable market in the U.S.

Naturally, there is the very real risk of clinical and regulatory setbacks. Also, gene-editing medicines aren't exactly mainstream in the healthcare community yet, and there are some roadblocks to mass adoption, including their typically high prices and the fact that they are often very difficult to administer. All of these are risks worth factoring in. With all that said, is the stock a buy?

My view is that CRISPR Therapeutics is a fairly attractive biotech company, given its deep gene-editing pipeline that features several potential breakthrough medicines, but only for investors comfortable with significant risk and volatility. The stock might soar through 2030 if it can advance key pipeline programs and avoid commercial pitfalls, but certain setbacks might sink the share price. Invest accordingly.

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.