CRISPR Therapeutics (CRSP 0.34%) has reached a very important chapter in its growth story. The gene-editing specialist doesn't have products on the market right now, but that could change in just a few weeks. The U.S. Food and Drug Administration (FDA) is set to decide on exa-cel, the company's gene-editing candidate for sickle cell disease, next month.
An approval could be the first step toward blockbuster status for the candidate. I say "first step" because regulators will decide on exa-cel as a treatment for a second blood disorder -- beta thalassemia -- in March. And CRISPR Therapeutics and partner Vertex Pharmaceuticals also are working on trials to expand use of the potential therapy to younger patients. Does all of this mean now is the time to buy CRISPR Therapeutics? Let's find out.
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CRISPR gene-editing
First, a bit of background on CRISPR Therapeutics and its technology. The company uses a process called CRISPR -- short for "clustered regularly interspaced short palindromic repeats" of genetic information -- to fix faulty genes responsible for disease.
The technology, based on a system used by bacteria to defend themselves from a virus, involves cutting DNA at a specific location and letting a natural repair occur.
What's particularly interesting about this technique is that it not only treats a disease, but through correcting defective genes, it also creates a functional cure. So CRISPR-based treatments could be game changers, especially since they address serious diseases that often have limited treatment options.
And this brings us back to exa-cel, designed as a one-time curative treatment for sickle cell disease and beta thalassemia. Doctors and patients might flock to it for the two points I mention above: its potential to cure their illnesses and the fact that there aren't many compelling therapies out there to address these blood disorders. And patients generally face a lifetime of hospitalizations.
Short personal story: My mother, who was a nurse, got to know her sickle cell patients well, following them from childhood through adulthood as they came in and out of the hospital. So, if exa-cel works over the very long term, it could truly be life-changing for patients.
An FDA advisory committee discusses exa-cel
Of course, gene editing is a newish, cutting-edge technology, so regulators are closely examining potential problems. An FDA advisory committee even met earlier this week to discuss the risk of off-target edits to the genome, which could lead to unwanted side effects.
Unlike most advisory committee meetings, this one didn't include a vote on the point discussed. But the panel's discussions about exa-cel's safety seemed favorable, a positive step along the road to a possible approval.
CRISPR Therapeutics and Vertex also have proposed an ongoing study, following patients for 15 years to monitor safety over the long term.
All of this means things are looking bright for exa-cel, and there's reason to be optimistic about upcoming regulatory decisions.
Now, let's get back to our question. Does this mean it's time to buy CRISPR Therapeutics? Yes -- but not for the reason you might think. I wouldn't buy this biotech with the idea that the stock will soar following an FDA decision. Potential good news could already be baked into the shares, and even if it isn't, one short-term move won't necessarily make or break your investment.
Long-term investing
Instead, it's best to focus on the long term. And from this perspective, I would buy CRISPR Therapeutics now because we've reached a great time to invest in the stock. The company is at the beginning of its earnings growth story, and as product revenue starts rolling in, we could see the shares progressively climb.
I'm not talking exclusively about exa-cel revenue. CRISPR Therapeutics has an entire pipeline of candidates based on its gene-editing technology. And another one, immuno-oncology candidate CTX-110, isn't too far from the finish line. It's in a phase 2 trial that could support a regulatory request.
So, right now is the ideal moment to buy shares of this gene-editing specialist and potentially win big over the long haul.
