Where Will CRISPR Therapeutics Be in 10 Years if Gene Editing Fulfills Its Promise?

Technically sophisticated gene-editing biotechs like CRISPR Therapeutics (NASDAQ: CRSP) could become dramatically more capable over the next 10 years if their most ambitious plans come to fruition. If that happens, it'd likely make those who invest today much wealthier.

But where exactly will CRISPR Therapeutics be in 2036, given what it's doing today and what it wants to do tomorrow?

Image source: Getty Images.

What the company is building toward

Today, CRISPR has one approved medicine, Casgevy, for sickle cell disease and transfusion-dependent beta thalassemia. The biotech's collaboration partner, Vertex Pharmaceuticals, sells the medicine and takes 60% of the profit.

Its second-quarter 2026 revenue came to $10.2 million against a net loss of $91.2 million, driven mostly by research spending. The therapy is still a heavy lift to give someone, requiring cell collection, editing in a lab, chemical treatment to clear the bone marrow, and reinfusion at a transplant center. That burden is what the company is trying to engineer away.

The company's answer is to do the gene-editing process inside the patient's body instead, turning a logistically complicated workflow into an infusion of a standardized therapeutic product. The company assesses that change would expand the pool of addressable patients worldwide from 150,000 to 400,000.

Another technical bet, SyNTase editing, seeks to solve a different issue entirely by correcting a mutation in a living organism instead of switching a dysfunctional gene off. Many inherited diseases need a broken protein restored rather than simply deactivated, so having a tool that does gene sequence correction would substantially widen the universe of potentially treatable diseases.

The pipeline program using this form of editing, CTX460, which is intended to treat alpha-1 antitrypsin deficiency (AATD), entered its phase 1 trial this year. Phase 1 assets that eventually reach approval take an average of 10.5 years to get there, so by 2036, it should be fairly clear whether this tech will work as desired.

If it succeeds, it'll be a landmark for the entire discipline of gene editing and make CRISPR the owner of several different proven technologies, all of which could be used to develop more medicines, potentially including combination therapies utilizing multiple gene-editing approaches at the same time for superior effect.

How things could play out

Let's now turn to three possible scenarios for CRISPR's position in 2036, assuming that its ambitions regarding gene editing are largely fulfilled.

In the least positive scenario, some of CRISPR's programs end up hitting the same problem that the big pharma company Novartis just recently ran into with one of its own lipid-lowering candidates.

In short, it's possible for a gene-editing therapy to succeed in its editing capacity, causing a patient's biomarkers to show a commensurate improvement, and then for the desired therapeutic outcome, like preventing heart attacks in Novartis' case, to never materialize. Worse, given the nature of CRISPR's line of business, developing gene-editing therapies, it could have that same problem multiple times in different disease contexts. This scenario would still see the stock priced higher than it is today, as the underlying technology would have multiple new proofs of its effectiveness, and the business would still likely have at least one or two new therapies approved for sale, but its growth trajectory might be a bit underwhelming overall.

The base case for the biotech would see two or three new approvals, like perhaps with its CTX310 program for severe hypertriglyceridemia, which is in phase 1b. The program's addressable population runs above 1 million U.S. patients, counting severe hypertriglyceridemia alongside refractory cholesterol disorders, and that sum might be even higher in 10 years. If a few new approvals happen, the stock will probably be worth a few multiples of what it's worth today.

The most bullish scenario would occur if the business were able to develop precise stem cell editing techniques to work in living people. This is where the promise of gene editing as conveyed in science fiction would start to look potentially attainable, and it's also where it's the hardest to predict exactly where the upside for investors would end. Though this scenario would require the company to make significant progress in its early pipeline over the next seven years or so, it's entirely possible.

So, given these possibilities and understanding that the reality might be a lot more mundane, is this stock worth buying?

If you're willing to hold it for the next decade or so, it's certainly worth a shot; CRISPR Therapeutics has a high potential to aim for the moon, fall short, and end up among the stars.

Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Vertex Pharmaceuticals. The Motley Fool recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy.