Hello, InvestorPlace Readers! This is the second installment of our new “How to Invest” series. You can see the first here: The Ultimate Guide to Investing in Growth Stocks. We’ll be covering investment opportunities ranging from genetic editing (see below) to electric vehicles to SPAC IPOs and much more. If you’re interested in a particular sector, please drop us a note to make a suggestion: email@example.com.
In June 2012, two scientists changed the world. U.C. Berkeley professor Jennifer Doudna and Umea University professor Emmanuelle Charpentier unveiled a radical scientific breakthrough — CRISPR-Cas9 genetic editing. This achievement would pave the way for a new niche of biotech stocks, dubbed CRISPR stocks.
Why is that important? Genetic editing’s widespread emergence and application in the 2020s will be one of the most transformative megatrends of our lifetimes.
And CRISPR stocks levered to this revolutionary megatrend will end up some of Wall Street’s biggest winners this decade.
And it all starts right now.
In short, Doudna and Charpentier figured out a time- and cost-effective way to manipulate genetic materials and, to some extent, “play God.” What’s followed over the past decade has been relentless innovation in the emerging field of genetic editing. Already, studies in this field have yielded game-changing results, such as the discovery and remediation of global vitamin A deficiencies.
And over the next decade, CRISPR-Cas9 genetic editing systems will become a major disruptive force. They will, quite literally, alter our reality.
They will help cure disease, fix genetic disorders, create sustainable biofuels, engineer pest-resistant crops and make life significantly better.
It may sound complex (and it is). But here, we will break down CRISPR-Cas9 systems, the business of genetic editing and why investors should care.
So, first things first: What exactly are CRISPR stocks?
What Is CRISPR-Cas9?
For decades, humans have been hyper-focused on genetic engineering and editing.
The discovery of the double helix in the 1950s and explosion of gene editing in the ‘60s set us on the path that biology and genetics are on today.
But the concept of rewriting genes themselves has always fallen short in practice. Developments like gene splicing and recombinant DNA moved the science forward, but they also posed an ethical hazard.
Instead of breeding two animals and hoping for the best, CRISPR-Cas9 allows scientists to rewrite entire genomes, creating unheard-of combinations.
Here’s the story. CRISPR’s principles were first pioneered by Francisco Mojica in 1993 as he worked closely with bacteria. Yes, the most transformative scientific breakthrough of our lifetimes — and maybe ever — comes from bacteria.
Mojica’s discovery sparked a chain of advancements in the CRISPR world, including the infamous “glo-fish” pets. They built off of the fluorescence naturally occurring in fish to create sub-generations that quite literally glow in the dark.
By 2012, Doudna and Charpentier advanced CRISPR to its most critical breakthrough — making precise, targeted cuts in DNA using “scissors,” or CRISPR-Cas9 systems. These scissors could be used to cut specific DNA threads and modify genes inside of living organisms.
Doudna and Charpentier noted a naturally occurring genome editing system in bacteria. Specifically, it had the odd ability to capture snippets of DNA from invading viruses. They then leverage that captured snippet to create a new DNA segment called a CRISPR array.
That CRISPR array is designed specifically to recognize the DNA of the invading virus. So, if/when the invading virus attacks the bacteria again, the array recognizes the DNA of the virus. It promptly deploys a Cas9 enzyme to “cut” the invading DNA segment, which renders the virus ineffective.
It’s a genius defensive mechanism that bacteria employ to ward off invading viruses.
Doudna and Charpentier wondered if they could replicate this brilliant genomic editing system in bacteria and use it elsewhere.
And they did just that.
Essentially, the two invented a way to engineer CRISPR arrays paired to a target sequence of DNA in a genome. That array is then injected into the genome, where it binds to the target sequence. Then, it uses a Cas9 enzyme to cut the DNA at the targeted location.
Once the DNA is cut, researchers can insert, remove, and/or edit genetic material in that DNA sequence however they please.
The Potential Implications of Genetic Editing
If that sounds like a big deal, that’s because it is.
At scale, the potential implications of genetic editing are infinite.
Doudna and Charpentier’s breakthrough discovery has since ushered in a new era of genetic engineering. And now some of the smartest people of the most prestigious labs are using CRISPR-Cas9 to potentially change the world.
Right now, research is being done to advance the use of CRISPR-Cas9 to cure diseases like cystic fibrosis, cancer, and sickle cell disease. CRISPR has the potential eradicate hereditary blindness. It’s being used to enhance biofuel tolerance (and in turn, create more sustainable biofuels). And researchers are also harnessing it to engineer more robust pest- and disease-resistant crops.
In other words, CRISPR-Cas9 is allowing us to create a better tomorrow without sickness, food shortages and resource depletion.
And so, I reiterate: The potential implications of CRISPR-Cas9 genetic editing systems are infinite. While important, this shouldn’t be surprising. After all, every living thing in this world has DNA. So, theoretically, a perfect CRISPR-Cas9 system could change anything about anything.
But, if true, why aren’t CRISPR-Cas9 systems everywhere? Why hasn’t cancer been cured? Why are crops still dying from pests and disease?
After all, CRISPR-Cas9 systems were unveiled eight years ago. Where are the real-world results?
The answer: Stuck behind an insurmountable mountain of genetic data.
And that’s where step two comes into play.
CRISPR Stocks and the Genetic Data Challenge
CRISPR-Cas9 genetic editing is a complex science. After all, you’re cutting DNA sequences. And to do that effectively without producing unwanted side effects, it requires great precision.
Imagine rearranging T.S. Elliot’s The Wasteland. A single change in the structure of the prose would change the entire meaning of the work, rendering it ineffectual. One stanza’s removal has an effect not just on the following or the preceding stanza but the entire work.
The same is true when it comes to cutting DNA sequences. If researchers are going to cut a DNA sequence precisely and effectively, they need to know everything about that sequence. What’s in it? What’s next to it? What are we knocking out? What will happen when we knock that genetic material out? Will there by side-effects when we do this process? How do we control those side effects?
It’s unlike cutting paper. A genetic editing process gone wrong could result in seriously negative side effects.
Cutting Through the Data
While researchers today are no less certain of their work than before, they’re now able to be more precise and advance the science more quickly.
Certainty is achieved through data. If researchers have all the DNA sequencing data on all genetic things in the world, they know exactly where and when to cut, what to inject and how to patch it all up when they’re done.
Unfortunately, that’s a big “if,” which has proven an enormous obstacle to effectively administering CRISPR-Cas9 systems. And it’s ultimately mostly kept genetic editing in the “hypothetical testing” phase thus far.
The human genome alone is made up of 20,000 genes and more than 3 billion base pairs. And everyone is different, as is every animal, every crop. Everything is different.
You get the picture. The enormity of genetic data in the world is unparalleled. Up until recently, scientists simply had no way to effectively comb through and understand all that data.
Then artificial intelligence (AI) showed up to the party.
The AI Breakthrough in Genomic Editing
Essentially, recent breakthroughs in AI have enabled machines and algorithms to comb through genetic data time- and cost-effectively. And that’s greatly helped researchers to make sense of all the data at their fingertips.
Case in point: Thanks to ingesting data from over a billion mutational outcomes from CRISPR-Cas9 systems, researchers at the Wellcome Sanger Institute have created a machine learning (ML) model that can effectively predict the exact mutations from CRISPR-Cas9 cuts using just the sequence of the target DNA as an input. That has enabled researchers to prevent unwanted “off-target” effects of such genetic engineering and/or remedy such effects quickly.
Another example: A research team led by Stephen Kingsmore of Rady Children’s Institute for Genomic Medicine paired a machine learning model called MOON with patient-specific information and data on more than 13,000 known genetic diseases in scientific literature. By doing so, they created an AI system that can identify likely disease-causing mutations out of 4.5 million potential variants. And that’s all in five minutes or less.
Yet another example: Researchers at the University of Copenhagen have developed a machine learning algorithm that recognizes protein movement patterns and classifies genetic data in seconds. This is a process that used to take the researchers several days to accomplish.
I could go on and on. But you get the point.
Dozens of AI/ML models have popped up over the past few years. And they’re enabling scientists to turn the complex and timely concept of CRISPR-Cas9 genetic editing systems into a disruptive reality.
The Economic Value Behind CRISPR Stocks
Make no mistake. This transition toward tangible genetic editing will happen on a widespread scale over the next decade. As it does, humans will take a huge leap forward in regard to curing disease, improving health, fortifying food safety. The list goes on and on.
As CRISPR stocks become more “mainstream,” the economic implications of this tectonic shift are enormous.
On the disease front, researchers have identified over 6,000 genetic disorders. Globally, they affect about 1 in every 50 people. CRISPR-Cas9 genetic editing systems have the potential to cure every single one of those disorders. And in so doing, they’ll entirely replace the $50 billion genetic disorders drug market. In that vein, the addressable market for genetic editing in curing human disease alone is worth over $50 billion.
Meanwhile, market research firms peg the global biofuels market as being worth north of $100 billion today and somewhere around $150 billion within the next four to five years.
Genetic editing could be the mechanism that unlocks even more significant value in that $150 billion and rapidly growing market.
Not to mention, the crop protection market measures over $60 billion globally. Genetic editing could flip that entire market on its head, too.
When you add it all up then, genetic editing systems have an addressable market that swells well over $200 billion.
Yet, most genetic editing companies today have a market cap of less than $5 billion. And this discrepancy between current valuations and long-term economic potential is your opportunity.
Five CRISPR Stocks to Watch
Finally, we are ready to answer the most important question. What are the best genetic editing stocks to buy to play the biggest scientific breakthrough of the century?
Here are a few genetic editing stocks that should be on you radar:
CRISPR Therapeutics (NASDAQ:CRSP) — the “mothership” of CRISPR-Cas9. Founded by Emmanuelle Charpentier — the French microbiologist who co-invented CRISPR-Cas9 systems back in 2012 — CRISPR Therapeutics is the largest and most well-established genetic editing company in the world. CRISPR is backed by the most robust and mature genetic editing therapy pipeline in the industry. That pipeline is mostly focused on curing sickle cell disease (with its CTX001 drug candidate that is in clinical trials) and delivering immuno-oncology cell therapy (with its CTX 110, CTX120, and CTX130 programs, all of which are also in clinical trials). The company also has a diabetes treatment in the pipeline with significant long-term potential. If you’re looking for a “blue chip” play on CRISPR stocks, CRSP is the best choice.
Editas Medicine (NASDAQ:EDIT) — the blindness doctor. This company is another large and well-established genetic editing firm, with a focus on the ocular world. Specifically, it has a portfolio of gene-editing therapies (EDIT-101, EDIT-102, and RP4) which together aim to eradicate inherited retinal diseases (i.e. genetic blindness). EDIT-101 is the most advanced of these therapies and is currently in Phase 1/2 clinical trials. Also of note, in December 2020, the company submitted an Investigational New Drug (IND) application with the FDA for the initiation of a Phase 1/2 clinical trial for its new EDIT-301 therapy for the treatment of sickle cell disease. And in March 2022, it received FDA orphan drug designation for EDIT-301. And it’s on track to dose the first patient by 2022’s end. If you’re looking for a more-focused genetic editing play with a good risk-reward balance, EDIT stock is a great choice.
Intellia Therapeutics (NASDAQ:NTLA) — the specialized gene editor. Founded by Jennifer Doudna — the other co-inventor of CRISPR-Cas9 systems — Intellia Therapeutics is a more specialized gene editor with a unique and potentially profound gene therapy delivery platform. Only one of the company’s gene therapies (NTLA-2001) is in clinical trials. So, the company is a bit “behind the curve” on the commercialization front. But all three of its therapies (NTLA-2001, alongside NTLA-2002 and NTLA-5001 — both of which submitted INDs in 2021) are attacking competition-less end markets like curing Hereditary Angioedema, Acute Myeloid Leukemia, and Transthyretin Amyloidosis. On top of that, Intellia built its therapies on a proprietary LNP delivery technology that’s shown a great number of benefits in non-human primate studies. If you’re looking for a more specialized gene editor with potentially explosive upside, NTLA stock is a solid pick.
Beam Therapeutics (NASDAQ:BEAM) — the “base” gene editor. This is a differentiated genetic editing company. It utilizes a unique approach called “base” editing to modify genetic structure. Instead of entirely cutting open DNA strands using Cas9 “scissors,” Beam chemically changes one DNA base in a genetic sequence to another, without completely cutting the backbone of the DNA. The result is a highly specific gene editing tool with substantially lower risk of off-target effects. Thus, while Beam’s therapy pipeline is very early stage relative to others (no drugs are in clinical trials yet), the company’s differentiated and potentially breakthrough approach implies that it could one day be the biggest genetic company in the world. So, if you’re looking for a more high-risk, high-reward genetic editing play, then BEAM stock could be your answer.
Cellectis (NASDAQ:CLLS) — the cancer killer. The genetic editing company focuses exclusively on curing cancer. Specifically, Cellectis is a long-time leader in using TALEN gene editing technology — a fundamentally different gene editing technology from CRISPR-Cas9 — to eradicate cancer cells. The company currently has three branded therapies in Phase 1 clinical trials. And it also has another three partner therapies in Phase 1 clinical trials, too. If you’re looking for increased exposure to genetic editing outside of just CRISPR-Cas9 techniques, CLLS stock could be the right pick.
Bottom Line on CRISPR Stocks
Imagine a world where you can identify individual genes you don’t want, and replace them with ones you do. For the millions of people suffering incurable genetic disorders from sickle cell to Tay-Sachs, that would mean years of added life. For farmers, that would mean drought and pest-resistant crops. And for humankind, it would be a triumph of science.
The convergence of artificial intelligence, DNA sequencing and genetic editing is going to change our world in an enormous, almost-unthinkable way. Disease will be cured. Genetic disorders will be eliminated. Crops won’t die from pests or disease anymore.
Life will get substantially better.
And it all starts right now.
On the date of publication, Luke Lango did not have (either directly or indirectly) any positions in the securities mentioned in this article.
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