CRISPR (clustered regularly interspaced short palindromic repeats) genome editing has been hyped for years. But the promise of CRISPR technology has yet to be fulfilled. That could change in the coming years, though. Major genomics breakthroughs — and potentially explosive growth for some stocks — could be on the way.
CRISPR companies forging the future
CRISPR companies forging the future
Several companies are using CRISPR to edit human genomes in an attempt to treat (and even cure) genetic diseases. Their therapies either use an ex vivo approach or an in vivo approach. With ex vivo therapies, genes are edited outside the body.
But CRISPR's use isn't limited to only genetic diseases. Several gene-editing companies are also targeting various types of cancer. In particular, CRISPR is being used to develop chimeric antigen receptor (CAR) T-cell therapies where immune cells are genetically engineered to attack specific tumors.
In addition, some companies are using CRISPR for screening (which shouldn't be confused with genetic testing). With CRISPR screening, genes are edited in a way that makes them inoperative. CRISPR screening enables companies to discover which genes perform specific functions, which can be important in developing drugs.
5 best CRISPR stocks to buy
5 best CRISPR stocks to buy
Not every investor will want to buy CRISPR gene-editing stocks. These stocks tend to be highly risky and volatile. However, for aggressive investors who aren't risk-averse, here are five of the best CRISPR stocks to consider:
| Company | Market Cap | Key Therapeutic Areas of Focus |
|---|---|---|
| Beam Therapeutics (NASDAQ:BEAM) | $1.8 billion | Sickle cell disease, alpha-1 antitrypsin deficiency, glycogen storage disease 1a, T-cell leukemia/lymphoma |
| CRISPR Therapeutics (NASDAQ:CRSP) | $4.1 billion | Beta-thalassemia, sickle cell disease, cancer, cardiovascular diseases, diabetes |
| Editas Medicine (NASDAQ:EDIT) | $276 million | Sickle cell disease, beta-thalassemia |
| Intellia Therapeutics (NASDAQ:NTLA) | $2.1 billion | Transthyretin (ATTR) amyloidosis, hereditary angioedema, alpha-1 antitrypsin deficiency-associated lung disease |
| Verve Therapeutics (NASDAQ:VERV) | $509 million | Cardiovascular diseases |
Beam Therapeutics
1. Beam Therapeutics
Beam Therapeutics' base-editing approach enables the company to rewrite a single letter of the genome. While other CRISPR methods are similar to genetic scissors, base editing is more like a pencil with an eraser.
The company believes that its approach offers several advantages compared to other CRISPR techniques. These include the ability to precisely target a specific gene sequence and edit genes without unintended consequences such as rearranging the genome on a larger-than-planned scale.
Beam isn't as far along as some of its CRISPR-focused peers. The company is evaluating four candidates in phase 1/2 clinical testing targeting the rare genetic disorders sickle cell disease, alpha-1 antitrypsin deficiency (AATD), and glycogen storage disease 1a (GSD1a) as well as T-cell leukemia/lymphoma.
The company's market cap isn't far below the levels of other CRISPR stocks with more advanced pipelines. However, the long-term potential for Beam's technology could justify this premium valuation.
CRISPR Therapeutics
2. CRISPR Therapeutics
CRISPR Therapeutics is the only CRISPR-focused biotech stock that has advanced beyond clinical-stage development. The company and its partner Vertex Pharmaceuticals (VRTX -2.42%) won U.S. regulatory approvals for Casgevy in treating sickle cell disease in December 2023 and transfusion-dependent beta-thalassemia in January 2024.
It's evaluating two experimental CAR T-cell therapies in clinical trials. CTX112 targets CD19+ B-cell malignancies. CTX131 targets solid tumors and blood cancers. Other pipeline programs for CRISPR Therapeutics include CTX310 and CTX320, which target cardiovascular disease, and CTX211 for treating type 1 diabetes.
The drugmaker also is exploring the use of CRISPR in several preclinical programs. It's researching in vivo therapies targeting rare genetic diseases, including cystic fibrosis and Duchenne muscular dystrophy.
Although all CRISPR stocks are risky, CRISPR Therapeutics is arguably less risky than most. It has a strong cash position, thanks mainly to its partnership with Vertex, with an approved product on the market.
Editas Medicine
3. Editas Medicine
Editas Medicine was leading the way in using CRISPR to treat rare genetic eye diseases. However, the company eventually threw in the towel on these efforts following disappointing clinical trial results.
But Editas still has high hopes for its lead candidate, reni-cel (previously known as EDIT-301). It's evaluating the experimental therapy in late-stage studies as a treatment for sickle cell disease and in early-stage studies targeting beta-thalassemia.
The company believes that reni-cel could be a "best-in-class" treatment for both sickle cell disease and beta-thalassemia. Results from clinical trials so far have been promising.
Editas is also continuing to work on developing in vivo therapies. So far, though, the company hasn't advanced any additional in vivo programs into clinical testing.
Intellia Therapeutics
4. Intellia Therapeutics
CRISPR Therapeutics isn't the only CRISPR-focused biotech company with a big partner. Intellia Therapeutics is teamed up with Regeneron Pharmaceuticals (REGN 0.55%). The two companies are evaluating NTLA-2001 in a phase 3 clinical study in treating rare genetic disease transthyretin amyloidosis (ATTR) with cardiomyopathy. This experimental therapy is also advancing into another phase 3 study targeting hereditary ATTR amyloidosis with polyneuropathy.
Intellia also has a late-stage program that it fully owns. In October 2024, the company advanced NTLA-2002 into phase 3 testing as a potential treatment for hereditary angioedema, a rare genetic disease where individuals often experience severe inflammatory attacks in different organs and tissues.
The company's pipeline includes two early-stage candidates. NTLA-3001 targets AATD-related lung disease. Intellia and Regeneron are also working together to evaluate CRISPR gene-editing therapies in treating hemophilia.
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Verve Therapeutics
5. Verve Therapeutics
Verve Therapeutics is one of the two smallest of these five CRISPR stocks. That's because the company's pipeline isn't as far along as some of its peers.
Like Beam Therapeutics, Verve uses CRISPR base editing. But Verve focuses on cardiovascular diseases. Its lead pipeline candidate, VERVE-101, targets heterozygous familial hypercholesterolemia (HeFH), a common type of atherosclerotic cardiovascular disease (ASCVD). Verve and its partner, Eli Lilly (LLY -2.37%), are evaluating the experimental therapy in a phase 1b clinical trial.
The two companies also have another early-stage base-editing therapy targeting HeFH. VERVE-102 targets the PCKS9 gene as VERVE-101 does. However, it uses a different lipid nanoparticle delivery method.
Verve is arguably the riskiest of these five CRISPR stocks. However, its base-editing approach looks promising.
Frequently Asked Questions
Frequently Asked Questions
Which company is leading CRISPR?
Although several biotech companies have CRISPR gene-editing therapies in clinical development, only CRISPR Therapeutics (NASDAQ:CRSPR) and its partner, Vertex Pharmaceuticals (NASDAQ:VRTX), have an approved CRISPR product on the market.
What is the best CRISPR stock to buy?
It's difficult to identify the sole best CRISPR stock to buy. CRISPR Therapeutics (NASDAQ:CRSP) is arguably the safest pick since the company already has a product on the market. However, other clinical-stage CRISPR stocks could have greater upside potential over the next few years, albeit with greater risk.
Should you invest in CRISPR?
CRISPR stocks probably won't be a good fit for risk-average investors. However, aggressive investors willing to wait for pipeline programs to mature could find several CRISPR stocks attractive.
What company has the patent for CRISPR?
The ownership of CRISPR patents is somewhat complicated. The Broad Institute of the Massachusetts Institute of Technology and Harvard University owns the patent for the use of CRISPR in cells with a nucleus, which includes most living organisms. The University of California-Berkeley, the University of Vienna, and Emmanuelle Charpentier also owns patents to CRISPR technology. In addition, several biotech companies, including Beam Therapeutics (NASDAQ:BEAM), CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT), and Intellia Therapeutics (NASDAQ:NTLA) own CRISPR-related patents.
