Investing in healthcare stocks has its perks. Since medical services are always in high demand, investors can rest assured that the industry won't stop being relevant anytime soon.
Still, things evolve quickly in healthcare, so it's important to find stocks that can stay ahead of the curve or, at the very least, keep up with the changing nature of the sector. Plenty of corporations on the market can do so, one of which is CRISPR Therapeutics (CRSP 0.34%). Let's find out why this gene-editing expert is worth investing in for the long haul.
An important approval on the way
Gene editing is a set of techniques that allows scientists to make precise changes to an organism's DNA. If a defective gene causes a disease, editing the gene in question might be a solution to treating or even curing the illness. CRISPR Therapeutics' entire platform is based on this simple-sounding idea that is extremely complicated to pull off in practice. The approach is relatively novel, too.
While there are already gene-editing treatments on the market, there aren't yet any that use the CRISPR editing technique, which earned its creators a Nobel Prize in chemistry.
CRISPR Therapeutics is pushing for breakthroughs, and it might be inching closer to its first major one. Its leading candidate is exa-cel, a potential therapy for two blood illnesses: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The company developed it in partnership with biotech giant Vertex Pharmaceuticals (VRTX -0.06%).
However, it's important to highlight that CRISPR Therapeutics originally created exa-cel. As small clinical-stage biotechs often do, it entered a collaboration with a larger company with more funds to help push the program forward faster, and to mitigate the risks associated with a potential failure. Its efforts seem about to pay off.
Exa-cel is currently being reviewed for potential approval in various countries, including the U.S. Recently, the U.S. Food and Drug Administration gathered a committee of independent experts to discuss the potential approval of exa-cel in treating SCD. The meeting was largely positive; the experts expressed support for approval.
And given that there aren't many therapies for SCD on the market -- and the huge burden the disease poses for patients, their families, and healthcare systems -- everything points toward approval.
What is exa-cel's market opportunity?
The current arrangement between CRISPR Therapeutics and Vertex is that they'll split the profits and losses associated with exa-cel on a 40/60 basis -- with 40% of the profits going to the smaller company. No one knows how much a course of treatment for the therapy will cost, but it won't be cheap; gene-editing therapies never are. However, a cost of about $2 million has been suggested as a relatively fair one.
CRISPR Therapeutics and Vertex will initially go after 32,000 patients in SCD and TDT in the U.S. and Europe, with potential label expansions that could help them target many more than that. For now, let's stick with the 32,000 figure; that's a potential market opportunity of $64 billion for the two companies.
They'll face some competition, notably from Bluebird Bio's lovo-cel in treating SCD and Zynteglo in targeting TDT. Lovo-cel isn't yet approved but could earn the green light soon after exa-cel. However, Bluebird Bio no longer operates in Europe. Moreover, it doesn't have the funds of Vertex Pharmaceuticals.
That's why there's a good chance exa-cel could capture a solid portion of this market. Even 40% of it, or $25.6 billion, would be impressive. Here's the moral of the story: Exa-cel boasts blockbuster potential, an excellent sign for CRISPR Therapeutics and its shareholders.
Why CRISPR Therapeutics is a buy
Aside from exa-cel's prospects, there are deeper reasons CRISPR Therapeutics is a no-brainer stock for biotech investors. The company has a pipeline of several promising gene-editing treatments; it has already proven that it can develop groundbreaking therapies in this incredibly complex field; it has attracted the partnership of a biotech giant; and it should have the funds to make solid progress in the years ahead.
Those factors should lead to solid performance through the next decade. And with a market capitalization of just $4.35 billion, the stock appears to be understating its long-term potentiai.
