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Could CRISPR Therapeutics Become the Next Vertex Pharmaceuticals?

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Vertex Pharmaceuticals (NASDAQ: VRTX) is a great example of biotech success. The company has built a cystic fibrosis (CF) empire, bringing in billions of dollars in revenue annually, and now is even expanding into other promising billion-dollar opportunities. It recently won approval for a non-opioid pain treatment that could be a game-changer for both patients and the company down the road.

Of course, Vertex's success didn't happen overnight -- and the company even had to quickly shift focus after its initial drug, a hepatitis C treatment, didn't deliver lasting growth. Vertex then bet heavily on its CF research and scored a win: The company now has five commercialized CF treatments, including one approved early this year.

CRISPR Therapeutics (NASDAQ: CRSP) is a biotech that generally tackles specialties different from those of Vertex, though it has partnered with Vertex on certain programs. For example, using CRISPR Therapeutics' gene-editing platform, the two companies brought blood-disorders treatment Casgevy to market a little over a year ago.

So these two players know each other well, though CRISPR Therapeutics is in the earlier stages of its product commercialization journey. Could it catch up to its bigger biotech peer, and even become the next Vertex in the eyes of investors? Let's find out.

Members of a research team smile in a lab.
Image source: Getty Images.

Fixing genes involved in disease

Let's start by taking a look at the CRISPR Therapeutics story so far. The company focuses on CRISPR gene editing, a technique that repairs faulty genes involved in disease; it does this by cutting DNA at a specific location and allowing a repair process to happen.

As mentioned, this biotech and partner Vertex used the technology in Casgevy and won the first-ever regulatory approval of a treatment based on CRISPR gene editing. This shows that regulators recognize the technique's potential, and may be amenable to approving other treatments involving it in the future. And this is particularly important for CRISPR Therapeutics, since its pipeline revolves around CRISPR gene editing, with several compelling candidates for indications from type 1 diabetes to cardiovascular disease.

Trial data results and a product rollout

This year could be an important one for the company for a couple of different reasons. First, it expects to deliver updates across clinical programs, and if these are positive, they could act as a catalyst for share-price gains. The company says it should release updates on CTX112 in oncology and autoimmune diseases by the middle of this year, and updates on the CTX131 program in solid tumors and hematologic malignancies this year as well.