A lot of the hype about CRISPR gene editing has died down. But the tremendous potential in this approach to treating diseases remains. And several biotechs continue to work hard to make CRISPR gene-editing therapies viable treatment options for patients.
The case for CRISPR Therapeutics
The biggest reason to consider investing in any clinical-stage biotech stock is the potential in its pipeline candidates. For CRISPR Therapeutics, that potential is enormous.
CRISPR Therapeutics claims three clinical-stage programs -- more than any other biotech that's focused on developing CRISPR gene-editing therapies. The company is evaluating CTX001 in a couple of phase 1/2 clinical studies, one targeting beta-thalassemia and another targeting sickle cell disease. It also has a phase 1/2 clinical study underway on CTX110, an allogeneic CAR-T cell therapy, targeting cancers related to CD19+ malignancies.
Beta-thalassemia and sickle cell disease are genetic blood diseases that affect 60,000 and 300,000 newborn babies, respectively, each year. Neither disease has an effective treatment on the market. CRISPR Therapeutics' approach to treating the diseases by upregulating fetal hemoglobin holds a lot of promise.
The company's efforts to develop an allogeneic cell therapy could be an even bigger winner. There are a handful of cell therapies on the market already, but these products require patients' immune cells to be sent to an off-site facility where they are genetically engineered to target cancer cells, then sent back to infuse into the patient. This is a lengthy and expensive process. CRISPR Therapeutics hopes to use gene editing to modify healthy donors' immune cells, with an off-the-shelf product that can greatly reduce the costs and time required for treatment with cell therapies.
In addition to CTX110, the biotech has another cell therapy waiting in the wings. CRISPR Therapeutics plans to begin a phase 1/2 study of CTX120 targeting B-cell maturation antigen (BCMA) for treating multiple myeloma in the first half of 2020.
CRISPR Therapeutics' partnership with Vertex Pharmaceuticals is another big plus. Vertex is collaborating with the company on the development of CTX110. Thanks mainly to collaboration revenue from Vertex, CRISPR Therapeutics is sitting on a cash stockpile of nearly $630 million and won't have to worry about raising additional cash for quite a while.
The case for Intellia Therapeutics
Like CRISPR Therapeutics, Intellia is targeting rare diseases as well as developing cell therapies targeting cancer. While Intellia isn't as far along in development as CRISPR Therapeutics is, its market cap is only around one-fifth of the size of CRISPR Therapeutics.
Intellia doesn't have a clinical-stage candidate yet, but that should soon change. The biotech expects to file for approval by the Food and Drug Administration to begin clinical testing of NTLA-2001 in treating transthyretin amyloidosis (ATTR) by the middle of next year. Intellia is partnering with Regeneron Pharmaceuticals on its ATTR program.
The company is also targeting a couple of rare genetic diseases on its own. Intellia is researching using CRISPR gene editing to treat alpha-1 antitrypsin deficiency (AATD) and primary hyperoxaluria type 1.
Intellia and partner Novartis haven't disclosed much information about their efforts to develop allogeneic cell therapies and other cancer treatments using CRISPR gene editing. However, Intellia is making progress on its internal program to develop a cell therapy targeting acute myeloid leukemia (AML). The company appears to be on course to choose its first T-cell receptor (TCR) cell therapy candidate for AML by the end of this year.
Just one success out of these efforts could easily lead to blockbuster sales for Intellia. And while its programs are still only in very early stages, preclinical results have been promising.
Intellia should be in a good position to fund operations and development for at least another two years. The company had cash, cash equivalents, and marketable securities totaling nearly $296 million at the end of September 2019.
I think that both of these biotech stocks could be winners over the long run. But if I had to choose just one of them, it would be CRISPR Therapeutics.
In my view, CRISPR Therapeutics' pipeline is unmatched among CRISPR-focused biotechs. Although there are risks with its gene-editing approach, I'm cautiously optimistic that CTX001 will be successful in treating both beta-thalassemia and sickle cell disease. I also think that CRISPR Therapeutics will be one of several biotechs that develop effective allogeneic cell therapies.
My hunch is that the day will come when CRISPR gene editing is a commonly used approach to treating genetic diseases, cancer, and other diseases. And I think that CRISPR Therapeutics' products will be prominent in the mix of those CRISPR gene-editing therapies.