Predicting the future is never easy (at least not with any hope of being remotely accurate). But attempting to look 10 years in the future for an early-stage biotech is especially tough.
CRISPR Therapeutics (NASDAQ:CRSP) is about as early as you can get with early-stage biotechs. The company began its first clinical study only a few months ago. There are multiple paths that could unfold based on how this study and others progress. Where will CRISPR Therapeutics be a decade from now? Here are some possible futures for the up-and-coming biotech.
CRISPR Therapeutics' dream scenario would definitely include a resounding success for its lead pipeline candidate, CTX001. The biotech is currently enrolling patients in a couple of phase 1/2 clinical studies evaluating the gene-editing therapy in treating rare blood diseases beta thalassemia and sickle cell disease (SCD).
Both of these studies are scheduled to wrap up in 2022, although primary results should be available in early 2021. Assuming all goes well, CRISPR Therapeutics will advance CTX001 to registrational studies. And if those studies are successful, the biotech could have its first product on the market well before 10 years from now.
In this scenario, CRISPR Therapeutics would probably be generating revenue in 2029 of more than $1 billion with CTX001. Current treatments for beta thalassemia and SCD are very limited. Patients frequently require transfusions and hospitalization. CTX001 could be a lifesaver for thousands of patients each year.
But CRISPR Therapeutics could also have another product on the market in 10 years. The biotech plans to begin an early-stage clinical study of allogeneic CAR-T therapy CTX110 in the first half of 2019. Current CAR-T therapies require expensive and slow processes where patients' own T cells are genetically engineered to fight specific types of cancer. Allogeneic CAR-T therapies use genetically engineered T cells from healthy donors. These "off-the-shelf" therapies enable immediate treatment at lower costs.
CTX110 targets tumors that express the CD19 protein. CRISPR Therapeutics also has several other allogeneic CAR-T therapies in preclinical testing that target other tumors, including those that express BCMA and CD70. In a dream scenario, the biotech would leverage what it learns with CTX110 into positive studies for other CAR-T therapies and launch the most effective cancer treatments available. Success for its allogeneic CAR-T therapies could make CRISPR Therapeutics one of the hottest biotechs on the market in 2029.
Things could turn out much worse for CRISPR Therapeutics, though. There's a real possibility that safety issues could doom the company's underlying technology.
CRISPR Therapeutics named itself after the gene-editing approach that it has helped pioneer -- CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). With CRISPR, bacterial enzymes are used to target specific sections of DNA. Sequences in the DNA can be disrupted (inactivated), deleted, or replaced.
Two serious concerns have been raised about CRISPR. One is that there could be unintended "off-target" changes made when using CRISPR to edit DNA sequences. Another is that using CRISPR could increase the risk of cancer.
The nightmare scenario for CRISPR Therapeutics is that these or other issues cause the biotech's efforts to develop gene-editing treatments for rare diseases and allogeneic CAR-T therapies to flop. This would go beyond just one clinical study failure but would impact the biotech's entire approach to research and development. Should serious and unresolvable problems emerge with CRISPR gene editing, CRISPR Therapeutics might not exist 10 years from now.
A more likely future
My view is that the nightmare scenario for CRISPR Therapeutics is much less likely than the dream scenario is. That's not to say that new safety concerns won't arise with CRISPR. However, I don't think any of them will be total showstoppers.
On the other hand, CRISPR Therapeutics might not have a smooth path to success. The company already faced an FDA clinical hold on beginning clinical testing in humans CTX001, although this clinical hold was subsequently lifted. It won't be surprising for CRISPR Therapeutics to run into more bumps and hurdles as it advances its pipeline candidates.
I think that CRISPR Therapeutics will achieve success with CTX001. Its approach is basically to mimic naturally occurring genetic variants that enable some patients with beta thalassemia and SCD to have reduced and even no symptoms of the diseases. Because the genetics of the diseases are so well understood, I think it improves CRISPR Therapeutics' odds of clinical success.
My hunch is that the company will also accomplish its goal of developing allogeneic CAR-T therapies using CRISPR gene editing. And I think that this will present an even greater long-term opportunity for CRISPR Therapeutics than CTX001.
However, there's one aspect of the nightmare scenario that I suspect will occur sometime within the next 10 years: CRISPR Therapeutics won't exist. I don't mean that the company will fold up. Instead, I think that the biotech is likely to be acquired.
The obvious candidates for buying CRISPR Therapeutics are its two big partners -- either Bayer or Vertex Pharmaceuticals. Both Bayer and Vertex already own stakes in CRISPR Therapeutics.
My money would be on Vertex ultimately acquiring its gene-editing partner at some point down the road. There's a great fit between the two companies in terms of scientific focus. Vertex is also rapidly growing its cash stockpile that it will want to put to good use.
Will my predictions prove to be accurate? Check back in 2029.
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