This weekend, Intellia (NASDAQ:NTLA) came out with a press-release for the ages, which has NTLA stock soaring Monday. In the release, Intellia’s first-of-its-kind clinical data shows promising results for their in vivo — tested on living humans — genome editing therapy.
As a result of this news, Intellia’s stock is way up, and other genome editing stocks are seeing gains as well.
Since these companies share a commonality in the technology they utilize to manipulate genomes, it makes sense that their stocks move in harmony.
Intellia’s phase-1 clinical study demonstrated effectiveness in treating a rare hereditary disease caused by mutations in a specific gene related to vitamin A transportation throughout the body. Symptoms of this disease can include loss of sensation in one’s extremities and issues with the heart, kidneys, eyes and gastrointestinal tract. In many cases, a liver transplant may be necessary.
While Intellia’s treatment doesn’t cure the person affected, it does show promising results in preventing progression of the disease.
Why Intellia’s Treatment Is Better
CRISPR — a system used to modify genomes based on one utilized by bacteria — isn’t the first genome editing technology, but it’s particularly exciting because it is “faster, cheaper, more accurate, and more efficient than other existing genome editing methods.”
The goal of Intellia’s CRISPR-based therapy, NTLA-2001, is to enter the body, target the very specific mutation-causing protein and inactivate it.
NTLA-2001 is administered intravenously, which means no liver transplant is necessary. This already makes it a more enticing option, since it is far less intrusive.
But the benefits don’t stop there.
This method of treatment is also more effective. The results Intellia gathered from their clinical study showed that a a single dose of NTLA-2001 can slow and even reverse the targeted disease. That’s also more than can be said for previous forms of treatment for this disease.
On top of that, there were no serious side effects that resulted from editing patients’ genomes.
What’s the Catch?
The catch is that this study only involves six patients. And the effectiveness wasn’t uniform across the board for all of these patients.
Typical, non-CRISPR treatment for this specific disease is capable of reducing the levels of the problematic protein by about 80%.
NTLA-2001 had a range of effectiveness from between 52% to as high as 96%.
But the variability in effectiveness could possibly be attributed to participants not receiving the same dosage. And data from this study shows promise that the lower effectiveness corresponds to the smaller doses of NTLA-2001. Intellia plans to determine whether this is always the case in the future.
It also remains to be seen whether a single does of NTLA-2001 is all it takes to effectively treat the disease. It’s only been about a month since patients were treated, so it will be important to see how things play out for the treatment in the long-term.
The Bottom Line on NTLA Stock
Naturally, all CRISPR stocks move upward whenever one of the companies celebrates a breakthrough with the technology. They all share a common, life-changing thread.
And this first clinical study will be the first of many promising studies to come. Genome editing will be one of the most transformative megatrends in the next decade. The technology will disrupt the entire medical industry, as well as other industries.
We’ve been following the “genomic revolution” for a while now. In fact, if you had invested in NTLA stock after our recommendation earlier this year, you’d be seeing upward of 90% gains after this latest push in CRISPR stocks across the board.
The technology behind this company, and other companies in the genome editing space, is truly revolutionary.
If you’re curious about some of the genetic editing companies we personally have on our radar, check out our genetic editing stock investment guide. It covers genetic editing, especially as it relates to CRISPR systems, in additional detail, and it also highlights some companies worth following.
And if you’re interested in other stocks, I have more than 40 hypergrowth stocks that could score investors Amazon-like returns over the next months and years.
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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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