There's a short answer to whether CRISPR Therapeutics (CRSP +4.60%) could help you become a millionaire: Yes. However, that response makes little sense unless you understand why the company's prospects are so strong.
Here are five reasons why the company, which just went public in 2016, could make you a millionaire.
Exa-cel could fund enormous expansion for the company
CRISPR and Vertex Pharmaceuticals say they expect to complete their biologics license application early next year for exa-cel, to treat blood disorders beta-thalassemia and severe sickle cell disease. The talk is the one-dose therapy, which has so far been curative in trials, would cost $2 million per dose. While that potential price tag has engendered no small bit of controversy, even one far lower would certainly help fund continuing operations for CRISPR, which would get a 40% share of profits from the treatment.
NASDAQ: CRSP
Key Data Points
The companies said that 30,000 people could benefit from the therapy, if approved, in the United States and European Union. Multiply $2 million by 30,000 and that equals $60 billion, the immediate addressable market for the therapy. Using a 40% "share of profits" calculation gives $24 billion in revenue for CRISPR. . That's an over-the-top best-case scenario, but even a more conservative scenario would be wildly lucrative.
CRISPR would almost immediately go from a company that has lost a little more than $539 million over nine months to one that could be consistently profitable.
Competition is a good thing
CRISPR is just one of several companies that are utilizing the Crispr-Cas9 gene-editing technique that won the Nobel Prize for chemistry in 2020. The method, which can rewrite a gene to thwart a disease, is also being used by Intellia Therapeutics, Editas Medicine, Beam Therapeutics, and Caribou Biosciences. The plethora of start-ups will serve to sharpen each company's science and that will help CRISPR Therapeutics, which will look to build on what is only a little bit of a head start over its competitors.
CRISPR editing isn't a zero-sum game with only one winner, but the presence of so many companies will advance the science that can be used for so many different applications. Just as the recent advancement of some Alzheimer's therapies are benefiting the stocks of several companies that are focusing on Alzheimer's treatments, future successes by one CRISPR gene-editing company will show investors the potential in other such companies.
Forget exa-cel and look at the rest of the pipeline
CRISPR only went public in 2019, but the company already has 22 programs in its pipeline, of which 5 have reached clinical trials, spanning a broad spectrum of therapies, from immuno-oncology to regenerative medicine to hemoglobinopathies to in vivo approaches.
The company has already proven the effectiveness of its allogenic CAR-T therapies with more than 100 patients safely dosed with CRISPR's CAR-T cells across four trials so far in CTX110 (exa-cel) and CTX130.
CRISPR editing is a remarkably versatile platform
CRISPR (clustered regularly interspaced short palindromic repeats) editing can be utilized to treat a huge number of maladies. There are more than 7,000 diseases caused by genetic disorders and CRISPR editing has the potential to treat all of them. The companies have started by targeting genetic blood disorders such as sickle cell disease and beta-thalassemia, but CRISPR editing has the potential to treat and even cure a multitude of cancers and diseases.
Many diseases are more chronically managed through medicines. CRISPR editing has the potential to be a one-time curative treatment by fixing genetic diseases in a single dose. It also could be used to halt infections created by a foreign invader, such as HIV. The number of potential applications means that CRISPR's pipeline could expand exponentially.
While this is true for CRISPR's competitors, the company is already ahead, has $1.9 billion in cash, and could build on successful platforms to branch out into other therapy areas ahead of other companies.
The investors were right -- just too early and not patient enough
The stock is down more than 42% so far this year. With its shares so far down, that leaves plenty of room to make money -- provided, of course, the stock bounces back. There's plenty of reason to believe that while the company's prior rise was aided by a lot of hype, once the company gets its first therapy approved by the Food and Drug Administration (FDA), it will see a surge that is more justified. However, this stock isn't for investors without long investment horizons. Holders of this stock must be patient for the company to turn profitable.
Take a look at what happened when after decades of trial and error, the first mRNA vaccine hit the shelves. Moderna was at $23 a share on March 13, 2020. On Dec. 18, the day the company's COVID-19 mRNA vaccine was given Emergency Use Authorization by the FDA, the stock traded at a high of $176. Today, it trades for more than $200 a share.
That kind of leap isn't that hard to imagine for the first approved CRISPR-editing therapy and CRISPR, as well as its exa-cel partner, Vertex, stand to benefit as well as investors with those companies.
