Is this finally the year CRISPR Therapeutics (CRSP -6.31%) bounces back? The biotech has lagged the market in recent years, but shares are up 50% over the past three months and have climbed 42% since the start of 2025. Of course, this recent run isn't a good enough reason to buy the stock.
The question is whether CRISPR Therapeutics can maintain this momentum and perform well over the long run. Does it still have what it takes? Let's find out.
What's happening with CRISPR Therapeutics?
It's been almost two years since CRISPR Therapeutics earned approval for Casgevy, the first CRISPR-based gene-editing medicine to hit the market, which it developed in collaboration with Vertex Pharmaceuticals. Despite this major accomplishment, the mid-cap biotech has faced some challenges, including those related to the difficulty of administering Casgevy.
The therapy needs to be manufactured using patients' own cells, then reinserted back into them, a procedure performed only in qualified treatment centers. Still, the technology has immense potential -- Casgevy is a one-time functional cure for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), two rare and otherwise lifelong blood disorders.
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Now that CRISPR Therapeutics has demonstrated that its approach can lead to regulatory approvals, the company is seeking to replicate that success with other medicines. The company's pipeline contains several candidates in various therapeutic areas, including oncology, cardiovascular diseases, and endocrine disorders. The one that has been making the most noise recently is CTX310, a medicine that inhibits the action of a protein called ANGPTL3, which helps regulate LDL cholesterol. High doses of this cholesterol can lead to severe cardiovascular events.
In an ongoing phase 1 study, CTX310 led to a meaningful reduction in LDL cholesterol and triglycerides (a type of fat that can also be dangerous in high levels) in patients with several diseases for which lowering LDL cholesterol or triglycerides is key. Perhaps CTX310's recent progress has been a factor in CRISPR Therapeutics' strong performance this year, but what comes next?
The long-term view
Here's a crucial difference between some of CRISPR Therapeutics' existing pipeline candidates, including CTX310, and Casgevy. The former is an in vivo gene-editing therapy, which means using it doesn't involve the complex process of collecting patients' cells and manufacturing a medicine to reinsert into the patient. In vivo gene-editing medicines are therapeutic agents administered directly to the patient.
CRISPR Therapeutics' progress with CTX310, as well as some of its other candidates, could help the stock maintain its momentum. The complexity involved in ex vivo therapies like Casgevy makes them less appealing to investors.
True, there is still plenty of work to be done with CTX310, but CRISPR Therapeutics has a fairly substantial pipeline for a company of its size. Even if this one does not end up being successful, other programs might: CRISPR Therapeutics' CTX112, an investigational cancer therapy, has received the Regenerative Medicine Advanced Therapy (RMAT) designation in certain B-cell malignacies from the U.S. Food and Drug Administration, which, among other things, tells us that early evidence suggests it could be more effective than current standards of care for the diseases it targets.
At any rate, CTX310's early-stage results so far look promising. Furthermore, CRISPR Therapeutics should have sufficient cash on hand to fund the development of this medicine. It ended the first quarter with $1.86 billion in cash and equivalents. Investors shouldn't completely discount Casgevy, either. Although it's taking some time, the medicine should eventually make a meaningful contribution to CRISPR Therapeutics' financial results.
There are about 60,000 SCD and TDT patients in the geographies the company is targeting, and a single treatment course of the medicine costs $2.2 million. That sounds expensive, but let's remember that without a medicine like Casgevy, the affected patients would have to deal with these conditions for their entire lives, as well as having lower life expectancy because of them. What does all this mean for investors?
CRISPR Therapeutics' shares could soar on consistent positive clinical and regulatory developments. It's essential to keep the usual potential risks biotech companies face, including clinical setbacks. CRISPR Therapeutics has only one marketed product and is consistently unprofitable, so the risk here is higher than average. But for those who can stomach the volatility, initiating a small position now could lead to monster returns over the long run.
