In June 2012, the world changed forever – and you probably didn’t even notice.
It was in that month’s issue of the Science journal that U.C. Berkeley professor Jennifer Doudna and Umea University professor Emmanuelle Charpentier unveiled what the scientific community has since labeled the “scientific breakthrough of the century.”
What was this breakthrough?
CRISPR-Cas9 genetic editing systems.
Long story short, Doudna and Charpentier invented a pair of “scissors” – called CRISPR-Cas9 systems – that 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, wherein bacteria capture snippets of DNA from invading viruses and create new DNA segments called CRISPR arrays that are designed specifically to recognize the DNA of the invading virus. Thus, once the virus attacks the bacteria again, the CRISPR array recognizes the virus’ DNA, and deploys a Cas9 enzyme to “cut” the invading DNA, rendering the virus ineffective.
It’s pure genius.
Doudna and Charpentier figured out how to harness the power of this genius genomic editing system in bacteria, and use it for anything.
Essentially, the two invented a way to engineer small CRISPR arrays paired to a target sequence of DNA in a genome. That CRISPR array is then injected into the genome, where it binds to the target sequence, and 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 as they please.
If that sounds like a big deal, it’s because it is…
Doudna and Charpentier’s breakthrough discovery has since ushered in a new era of genetic engineering, wherein some of the smartest people in some of the most prestigious labs in the world are using CRISPR-Cas9 systems to potentially:
- Cure diseases like cystic fibrosis, cancer, and sickle cell disease.
- Create sustainable biofuels.
- Engineer more robust, pest- and disease-resistant crops.
- And much more.
Indeed, the potential applications of CRISPR-Cas9 genetic editing systems are endless.
Because every living thing has DNA, meaning that a perfect CRISPR-Cas9 system could theoretically change anything about anything.
But… if true… then why aren’t CRISPR-Cas9 systems everywhere? Why hasn’t cancer been cured? Why are crops still dying from pests and disease?
After all, it’s been eight years since CRISPR-Cas9 systems were invented. Where are the real-world results?
The answer: Stuck behind an insurmountable mountain of genetic data.
But that’s changing… right now… and as it does, the long-overdue genetic engineering boom will finally happen, and gene-editing stocks will soar several hundred percent.
Here’s the story…
CRISPR-Cas9 genetic editing is a complex science. You are, after all, cutting DNA sequences – which, to do effectively and without producing unwanted side effects, requires precision and accuracy.
Precision and accuracy are achieved through data. If researchers have all the data on all genetic things in the world, they know exactly where to cut, when to cut, what to inject after they cut, and how to patch it all up when they’re done.
Unfortunately, that’s a big “if,” which has proven to be an enormous obstacle to effectively administering CRISPR-Cas9 systems…
The human genome alone is made up of 20,000 genes and more than 3 billion base pairs. And everyone is different. Plus, every animal is different. Every crop is different. Quite literally, 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 of effectively combing through and understanding all of that genetic data.
Then artificial intelligence (AI) showed up to the party.
In essence, breakthroughs in artificial intelligence over the past few years have enabled machines and algorithms to effectively comb through and help researchers make sense of all the genetic data at their fingertips.
Case-in-point: Researchers at the Wellcome Sanger Institute have created a machine-learning model which – thanks to ingesting data from over a billion mutational outcomes from CRISPR-Cas9 systems – can effectively predict the exact mutations from CRISPR-Cas9 systems from just the sequence of the target DNA, thereby enabling researchers to prevent unwanted side effects of such genetic engineering.
Dozens of these of AI models have popped up over the past few years. They are slowly, but surely, enabling scientists to take the promising concept of CRISPR-Cas9 genetic editing systems, and turn them into a disruptive reality.
Make no mistake. This transition will happen on a widespread scale over the next decade. As it does, humans will take a huge leap forward when it comes to curing disease, improving health, fortifying food safety, and much, much more.
And guess what? This transition is starting right now – like, literally, right now.
This past Monday – so, three days ago – one of the leading gene-editing companies in the world, Intellia Therapeutics (NTLA), released interim data from a phase 1 trial of its CRISPR candidate in which Intellia showed that it was able to genetically edit cells inside a liver.
That’s game-changing news.
Previously, gene-editing was restricted to cells outside the body or in the eye. In-the-body gene-editing had never been done before. Until Monday. And now, a whole slew of doors just opened up for the gene-editing industry, wherein we can now apply gene-editing technology to anything.
You may have noticed. Gene-editing stocks jumped on the news.
But you haven’t missed out – because this rally is just the beginning of a multi-year breakout in gene-editing stocks as these companies go on to fundamentally change how we treat diseases.
So, what stocks should be buying to capitalize on the biggest scientific breakthrough of the century? I have 7 names on my watchlist:
- Intellia Therapeutics: The company that is pioneering the big breakthrough everyone is talking about, not surprisingly doubles as the one of the leaders in this space, having developed a portfolio of genetic editing therapies aimed at curing “rarer” diseases like Hereditary Angioedema, Acute Myeloid Leukemia, and Transthyretin Amyloidosis.
- CRISPR Therapeutics (CRSP): CRISPR was founded by Emmanuelle Charpentier – the French microbiologist who co-invented CRISPR-Cas9 systems. It is the largest and most well-established genetic editing company in the world, with a robust and mature gene-editing therapy pipeline focused on curing sickle cell disease and delivering immune-oncology cell therapies.
- Editas Medicine (EDIT): Editas has an ocular focus, with a portfolio of gene-editing therapies aimed at eradicating inherited retinal disease (i.e. genetic blindness)
- Beam Therapeutics (BEAM): Beam employs a unique genetic editing approach called “base” editing which is less intrusive than traditional CRISPR-Cas9 systems and possesses substantially lower risk of off-target effects.
- Cellectis (CLLS): Cellectics uses a non-CRISPR gene-editing technology called TALEN to eradicate cancer cells.
- Sangamo Therapeutics (SGMO): This company has a robust set of genomic medicines addressing multiple diseases – a well-diversified player in the space.
- Calayxt (CLXT): One of the more interesting plays in this space, Calayxt is using TALEN gene-editing technology to create a new generation of superior crops.
Between these seven genetic editing pioneers, you have the highest-quality and widest exposure to the biggest scientific breakthrough of the century.
Don’t this truly once-in-a-lifetime opportunity pass you up…
Full story on InvestorPlace.com
Leave a Comment