Genetic medicine is making waves. Here are some of the most exciting players.
- CRISPR/Cas is a new technology with the potential to cure genetic diseases, making certain CRISPR gene editing stocks to buy top investments.
- CRISPR Therapeutics (CRSP): Cofounded by a Nobel Prize winner and holds licenses to the main CRISPR patents.
- Beam Therapeutics (BEAM): Using a variation of CRISPR/Cas that promises to be more accurate.
- Vertex Pharmaceuticals (VRTX): Running clinical trials for what could be the first FDA-approved CRISPR/Cas drug.
The 2022 Nobel Prize in Chemistry went to Emmanuelle Charpentier and Jennifer Doudna for their work on developing tools to change the genetic code of living cells, tools called CRISPR/Cas. Yet long before their trip to Stockholm, they and other scientists had been diligently founding companies to monetize this new discovery. These companies bring new avenues in medicine and biotech that a smart investor can advantage.
CRISPR/Cas allows scientists to work on curing previously incurable genetic diseases. From Sickle Cell Anemia to Duchenne muscular dystrophy, patients are crying out for cures that CRISPR/Cas can bring. This has brought CRISPR Therapeutics (NASDAQ:CRSP) to the forefront as Charpentier cofounded it and it owns licenses to several CRISPR/Cas patents needed for drugs.
Alongside it, Beam Therapeutics (NASDAQ:BEAM) is using an even newer variation of CRISPR/Cas to create its own drugs. And established pharma companies like Vertex Pharmaceuticals (NASDAQ:VRTX) have entered the CRISPR/Cas business, hoping they’ll have the first FDA-approved CRISPR/Cas drug.
CRISPR/Cas is still very young, as are many of the companies monetizing it. That means it’s the perfect industry to invest in on the ground floor.
Here are three of the best CRISPR gene editing stocks to buy right now.
Data last updated: March 10, 2023 12:40 AM EST
|CRSP||Crispr Therapeutics Ag||$46.59|
|BEAM||Beam Therapeutics Inc||$35.21|
CRISPR Therapeutics (CRSP)
CRISPR Therapeutics (NASDAQ:CRSP) owns licenses to patents for a CRISPR/Cas system that can alter people’s genes and cure diseases. With Nobel Prize winner Emmanuelle Charpentier as one of its founders, CRISPR Therapeutics obviously knows its science. But it still needs to show that it can make consistent money.
CRISPR Therapeutics has already lined up collaborations. In 2021 it signed a deal with Vertex Pharmaceuticals for $900 million and 40% of the profits on a potential CRISPR drug called CTX001 to treat sickle cell disease and beta thalassemia. Current therapies for these diseases only mitigate the symptoms, but CTX001 hopes to be a cure. That could be a game-changer for patients and big money for CRISPR.
But that was 2021. In 2022, CRISPR reported just $1.2 million in total revenue and a net loss of $650 million. If CTX001 works, then regular revenue could follow. But if it doesn’t, CRISPR Therapeutics will need a new source of income… and fast.
CRISPR Therapeutics holds cash and securities worth $1.85 billion. So its current burn rate gives it three years of runway. CTX001’s clinical trials are in Phases 2 and 3, and slated to end in two or three years. Investors should look for results starting in late 2024.
Ultimately, CRISPR Therapeutics is a high-risk/high-reward play. FDA approval following clinical trials or new collaborations would bring in new revenue and send the stock soaring. But a bad clinical trial would put CRSP stock in a tight spot. Likewise, the company will need to show consistent revenue year after year to attract long-term investors.
Beam Therapeutics (BEAM)
Beam Therapeutics (NASDAQ:BEAM) uses a type of gene editing similar to the CRISPR/Cas called prime editing. Unlike CRISPR/Cas, which cuts both strands of the DNA, prime editing cuts just one strand. This more delicate technique should mean prime editing has less side effects than CRISPR/Cas. And like CRISPR/Cas, prime editing can be used to alter genes and cure diseases.
Like CRISPR Therapeutics, Beam has revenue but not consistency. In 2022 it entered into a collaboration with Pfizer (NYSE: PFE) which netted the company $300 million. The two will use Beam’s technology to target rare diseases. But aside from that, Beam’s annual report shows just $60 million in revenue for 2022 and a net loss of $289 million.
Beam has $1.1 billion dollars in cash and marketable securities. At its current burn rate, it can keep this up for at least four years. But to become profitable, it’ll need more collaborations and a lot more revenue.
That burn rate could be a problem, as Beam is a bit behind compared to CRISPR. It currently has just one active clinical trial, which is Phase 1/2 and slated to end in 2027. It will then need Phase 3 clinical trials before it can get FDA approval. Like CRISPR Therapeutics, Beam is targeting Sickle Cell Anemia. But if CRISPR’s drug comes out first, one wonders if Beam’s drug will find a market.
If you just want exposure to gene therapies, investing in both CRISPR and Beam makes sense. If you can only choose one, Beam is the riskier play. Investing in Beam over CRISPR is making a bet that the new technology can be overtaken by even newer technology. Investing in both is a bet that the future is bright for genetic medicine.
Vertex Pharmaceuticals (VRTX)
Vertex Pharmaceuticals (NASDAQ:VRTX) is an established drug company looking to have the first FDA-approved CRISRP/Cas drug. For 2022, Vertex’s annual report shows $8.9 billion in revenue and $3.3 billion in earnings. So it has plenty of room to grow into this business without fear of burning its limited cash.
As stated above, Vertex and CRISPR Therapeutics inked a deal to share profits on CTX001, a CRISPR/Cas drug for treating Sickle Cell Anemia and Beta Thalassemia. Sickle Cell Anemia is a popular CRISPR/Cas target because it’s difficult to get the CRISPR/Cas machinery into the human body and make it work. With blood diseases however, you can bring cells out of the body, modify them, and put them back in.
Unlike a patient’s blood, you can’t remove a patient’s muscle in order to modify it and put it back in. Once the low-hanging fruit of blood diseases is picked, companies will have a harder time with non-blood diseases like Duchenne’s. These diseases will require intense research into new delivery systems for CRIPSR/Cas. Vertex Pharmaceuticals has the cashflow and time to do this research.
Investing in Vertex will require a long-term outlook. CTX001 is undergoing multiple clinical trials with completion dates from late 2024 into early 2026. However the company itself is still a sound investment even without CTX001.
Vertex has plenty of room to work on CTX001 and its success could encourage plenty of new CRISPR drugs. That would be good for Vertex, and even better for CRISPR Pharmaceuticals.
This post originally appeared at InvestorPlace.
On the date of publication, John Blankenhorn did not hold (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.