The hunt for winning biotech stocks never truly ends, and it's easy to see why. Over the last five years, shares of the gene-editing company CRISPR Therapeutics (CRSP 0.34%) are up by 146%, easily topping the market's return of 94%. The company recently succeeded in its bid to commercialize a gene therapy for a pair of rare hereditary blood diseases.
As smart investors know, the heyday of gene editing has barely begun, so there's likely to be riches in store for those who can pick tomorrow's star performers. But the next great gene-editing stock won't be in the same vein as CRISPR Therapeutics, so what's the best contender to buy and hold right now?
My money's on Caribou Biosciences (CRBU -1.33%), and here's why.
This one isn't for the faint of heart, but the early signs are very positive
Caribou aims to treat relapsed or refractory (R/R) blood cancers like B-cell non-Hodgkin lymphoma (R/R B-NHL) and multiple myeloma (R/R MM) using its cell therapies. But its therapies aren't hindered by the pitfalls of yesteryear encountered by CRISPR Therapeutics and other competitors like Bluebird Bio.
Instead of using an obscenely complicated and weeks-long process that requires patients to donate their own cells as raw materials, one of the big innovations of the biotech's platform is that its cell therapies can be manufactured centrally, without any need for stem cell donations from patients. It'll save significantly on costs, and likely also create more effective medicines.
Caribou's lead candidate, CB-010 for non-Hodgkin lymphoma, has a trio of gene edits that will give it a major edge over competing products with fewer features, by making it safer and more effective. Per an update from the phase 1 clinical trial in July of 2023, 15 of 16 patients enrolled responded to treatment -- a remarkably good result. Most of the patients had an aggressive form of the disease, but six months after treatment, 69% of patients were still responding to treatment. In other words, preliminary data indicates that Caribou's candidate could be a game changer for patients who are desperately in need.
Regulators at the Food and Drug Administration (FDA) have already granted the company's request for the fast track, orphan drug, and regenerative medicine advanced therapy (RMAT) designations for CB-010. That suggests they see the program as a potentially helpful addition to the compendium of oncology medicines, which is a positive early sign.
Furthermore, as of the third quarter, Caribou had $397 million in cash, equivalents, and short-term investments. Management assumes that'll be enough to last through Q4 of 2025, at which point it'll need to find a research and development (R&D) collaboration partner, raise more funds, or both. If it can show more data of the same quality as its latest CB-010 release, it won't have any trouble surviving past that date.
On the other hand, it could potentially go to zero if it can't produce convincing data, so this is without a doubt a highly risky stock.
Can it actually perform as well as CRISPR Therapeutics?
On paper, Caribou Biosciences could potentially perform even better than CRISPR Therapeutics.
Whereas CRISPR Therapeutics targeted two rare diseases with relatively small markets -- beta thalassemia and sickle cell disease -- Caribou wants to fish in a bigger pond. The market for therapies that treat non-Hodgkin lymphoma was worth more than $9 billion in 2022, per Grand View Research, and demand is expected to rise at a rate of 8% annually between now and 2030. Furthermore, CRISPR Therapeutics' treatment may be curative for both illnesses, so its addressable market could shrink over time, but the same issue doesn't apply for the smaller company's candidate.
The catch is that Caribou is still early in its journey toward commercializing its first medicine, and it might not ever get there. As favorable as its phase 1 results are, late-stage clinical trial failures are the norm in biopharma, especially in the context of oncology, where failure rates are even higher than for other disease areas. Per some estimates, the probability of a cancer therapy surviving all the way from phase 1 trials to approval is in the ballpark of 3.4%.
So even though this biotech is my single most promising gene-editing investment, the odds are against it, and there hasn't been much in the way of de-risking as of yet. This isn't an opportunity you'd want to bet the farm on in hopes of creating generational wealth, as it's simply too risky.
Nonetheless, when it comes to biotech success stories, Caribou's auspicious start is a good omen. And at least for now, it looks like it has what it takes to be the next CRISPR Therapeutics.
