Where Will CRISPR Therapeutics Stock Be in 2030?

CRISPR Therapeutics (NASDAQ: CRSP), a gene editing-focused biotech, has made significant clinical and regulatory progress over the past five years. However, over this period, the company has lagged broader equities. CRISPR Therapeutics' shares have declined by about 47% since mid-October 2021, while the S&P 500 has gained almost 73%, as of writing.

Will CRISPR Therapeutics continue its southbound journey through the end of the decade, or does the company have what it takes to bounce back? Let's see where the biotech might be in 2030.

Pipeline progress will be key

In 2023 and early 2024, CRISPR Therapeutics earned approval for Casgevy, a gene-editing medicine for transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD), both of which are blood-related disorders. CRISPR Therapeutics collaborated with Vertex Pharmaceuticals (NASDAQ: VRTX) to develop this therapy. The approval was an important milestone for the company for at least two reasons.

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First, before Casgevy, CRISPR Therapeutics didn't have a single product on the market. This treatment allowed it to transition to a commercial biotech. Second, Casgevy was the first CRISPR-based gene-editing therapy to earn approval. Since most of CRISPR Therapeutics' pipeline candidates are CRISPR-based gene-editing medicines, Casgevy's launch lent more credence to the company's platform. CRISPR Therapeutics hasn't made much significant clinical progress since Casgevy's approval, but the company has several exciting pipeline candidates that investors should keep an eye on.

One of them is CTX310, a medicine being developed to help lower LDL cholesterol and triglycerides (TGs) in some patients. Why is this important? At high levels, LDL and TGs can cause a range of cardiovascular problems. Treatment options do exist, but they often need to be taken daily. CTX310 could be a one-time option to help some patients reduce LDL and TGs. CRISPR Therapeutics recently reported data from a Phase 1 clinical trial in certain patients with high levels of LDL or TGs, in some cases due to rare genetic disorders. The medicine led to significant decreases in both LDL and TGs.

It's still too early to celebrate, but over the next five years, CRISPR Therapeutics could make significant progress with CTX310 and perhaps earn approval, opening up a potentially highly lucrative opportunity.

CRISPR Therapeutics could also make meaningful progress with zugo-cel, an investigational medicine for several autoimmune diseases and blood cancers. Another pipeline candidate to keep an eye out for is CTX611, an investigational anticoagulant. CTX611 could be administered just twice a year, unlike today's anticoagulants, which are taken regularly. It is also being developed to reduce the risk of significant bleeding associated with many products in this niche. Perhaps the best-case scenario for CRISPR Therapeutics over the next five years would be to earn approval for all these medicines.

Hedge your bets

If CRISPR Therapeutics does launch several of its promising therapies by 2030, the company's financial results will improve significantly, and its share price will likely jump. In the meantime, Casgevy's sales could also ramp up. Earlier this year, the therapy earned a label expansion in treating TDT and SCD patients as young as two, which added about 5,500 patients in the U.S. to its target market. That doesn't sound like a lot, but at a price of $2.2 million per treatment course, that's a meaningful expansion.

However, there are significant risks to consider. CRISPR Therapeutics' leading pipeline candidates could fail in clinical trials. It wouldn't be the first time the company has experienced a clinical setback. Also, many gene-editing medicines -- including some that CRISPR Therapeutics is developing -- are one-and-done treatments that are costly to manufacture and administer. So, the economics of these therapies aren't obviously conducive to profits. CRISPR Therapeutics will have to overcome this challenge.

The biotech is, without question, highly innovative, and it may see its shares soar over the medium term on strong clinical and commercial progress. But there is significant risk, too. Only investors comfortable with that should consider initiating a position in the stock.

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.