Gene editing stocks are one of the hottest sectors in medicine today. Genes are the foundation of life, and from creating new organisms to curing incurable diseases, the editing of genes can bring radical changes to life as we know it. That’s why CRISPR stocks that can edit genes have been in such demand both in research and in the biochemistry sector.
But while gene editing can do many things, it can be hard to sort out science-fact from science-fiction. There are still many challenges that gene editing stocks face. And much research that still needs to be done. But that only means that the companies that get ahead of the pack can reap huge rewards. With so much on the line, many companies are creating exactly the kind of unbelievable results that you won’t want to miss out on.
Economically, gene editing is still in its infancy. Because of that, many companies in the sector are essentially pre-revenue. But while there may be a lack of discounted cash flow, there are still explosive growth opportunities. With so many products being developed for so many purposes, any one company could become a giant of the biotech industry overnight. The investors who get in before that happens can reap big rewards.
With all that said, here are the most exciting genomic stocks that you won’t want to miss out on.
Intellia Therapeutics (NTLA)
Intellia (NASDAQ:NTLA) was founded in part by Jennifer Doudna, co-winner of the CRISPR/Cas9 Nobel Prize. The company has since made a name for itself in studying and applying the CRISPR/Cas9 to curing incurable diseases.
One of the difficulties with gene editing is delivering the editing machinery to the right cells. CRISPR/Cas9 is a foreign entity to our bodies. And our bodies have evolved numerous mechanisms for destroying anything foreign that tries to enter. That’s why so many companies focus on blood-based diseases like sickle cell anemia or beta thalassemia. That’s because you can alter the DNA of a patient’s blood cells, then reinsert them into the patient without too much difficulty. You can’t do that with major organs, as removing, altering, and re-inserting them usually causes problems.
But Intellia has worked hard on that problem. They were an early pioneer of using Lipid NanoParticles for delivering CRISPR/Cas9 systems. And now their pipeline contains drugs for curing liver, lung, and other organ diseases. That means their potential market is much larger than the companies which are only focusing on blood-based diseases.
Intellia is still early, and none of their drugs have made it to the late-stage clinical trials which preface FDA approval. But they still have some runway to get their drugs approved. Their 2022 full year report shows they have about a year’s worth of cash and cash equivalents. And they have another year of marketable securities on their books. That means they still have some years to prove that their delivery system works. And I think they can do it.
Beam Therapeutics (BEAM)
Beam Therapeutics (NASDAQ:BEAM) founders are their own titans of the CRISRP/Cas9 community. David Liu, Feng Zhang and Keith Joung not only studied CRISPR/Cas9, they also studied a new form of gene editing called Prime Editing. Unlike traditional CRISPR/Cas9, which breaks both strands of DNA, Prime Editing breaks just 1 strand. Beam believes this technique has far more precision, and far less off-target effects. And that means that safer gene therapies may only be possible with Beam’s technology.
Beam Therapeutics has also had success in making the collaborations that will be necessary to bring its technology mainstream. They inked a deal with Pfizer (NYSE:PFE) to receive $300 million upfront, and a possible $1.05 billion in milestone payments in exchange for a 35/65 (Beam/Pfizer) split of net profits on upcoming Prime editing drugs. That could be a huge windfall for the company, as their annual net loss has hovered around $300 million these past years.
Still, Beam has the cash and assets to survive the near term. Investors should keep an eye on Beam’s furthest-along clinical trial, scheduled to reach its endpoint in 2025. They’re testing a technique to cure sickle cell anemia with prime editing. If that goes well, it could provide a boost of confidence for the stock, and herald much more to come.
Ultimately, investing in Beam Therapeutics over other gene editors is a question of the new versus the old. Gene editing may still be recent, but in a fast-moving field like this it can be brutal to keep up. If Beam’s technology is safer and more effective than the CRISPR/Cas9 systems used by competitors, it will have a decisive edge. And even if it’s only just as effective, Beam’s collaborations show they have the acumen to find a niche.
Editas Medicine (EDIT)
Another Jennifer Doudna co-founded company is Editas Medicine (NASDAQ:EDIT). Like Intellia, Editas is among the gene editing stocks using CRISPR/Cas9 systems to to solve incurable diseases. Editas has recently undergone a restructuring, selling off their cancer-cell therapy work to Shoreline Biosciences (privately held) for an undisclosed sum. But this should allow them to focus on their sickle cell anemia and beta thalassemia drug pipeline.
Editas is working on the most heavily-studied area of gene editing. By removing a patient’s blood cells, then altering them and returning them to a patient, many blood-based diseases can be cured. But that isn’t necessarily a downside, as Editas’ products are probably a safer bet than the more esoteric targets of other gene editing drugs. Because the cells can be altered in a controlled lab environment, any mutations can be screened and removed before infusion. And that means less off-target effects and more patient safety, twin pillars that are needed for FDA approval.
Of all the genomic stocks listed here, Editas may have the shortest runway. Their 2022 year-end report (from before the Shoreline deal) showed them with cash and securities worth about 1 year of runway. But the windfall from the Shoreline deal means they have more runway and less costs as they focus on their core drugs. As they’re working on becoming the most proven play in gene editing, they should definitely be looked at as one of the most promising genomic stocks on offer.
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.