You may have noticed several genetics companies skyrocketing this week with double digit gains every day following a major catalyst that took place over the weekend. That catalyst was reported by Intellia Therapeutics (NASDAQ:NTLA) along with its partner for the treatment and clinical trials, Regeneron (NASDAQ:REGN). But what was so significant that it led to Intellia basically doubling on Monday morning?
I’m no scientist or doctor, so if this sounds overly simplistic and very much unscientific, you’ve been forewarned. Intellia and Regeneron achieved the first ever successful in vivo CRISPR gene editing to ostensibly cure the disease known as transthyretin amyloidosis. That’s a mouthful, but I’ll try and break this down so it’s a little easier to understand.
This sounds like something out of a science fiction movie, but essentially Intellia has discovered a way to send lipid nanoparticles with the CRISPR gene editing instructions inside, to the patient’s liver. Once they arrive at the liver, the process of gene editing begins where the strands of DNA that cause transthyretin amyloidosis are effectively cut out, which stops the liver from creating the harmful proteins. It’s been called a groundbreaking moment for modern medicine, and the average success rate of 87% in the Phase 1 clinical trial for the patients who were given a higher dose is truly incredible.
While transthyretin amyloidosis or ATTR for short, only affects about 50,000 patients around the world, the broader gene editing science is the real takeaway here. Once this is perfected, the treatment can remove those radical strands of DNA that cause genetic diseases like sickle cell. This could potentially wipe out hundreds of genetic diseases from the face of the Earth.
There are some drawbacks of course. It was a Phase 1 clinical trial, so let’s see what the results are from Phase 2 and 3 before we draw final conclusions about the treatment. CRISPR gene editing is also still an extremely expensive treatment that is not accessible by many hospitals or institutions at the moment. Finally, the shipment of these nanoparticles and CRISPR instructions will have to be tested for other organs in the body, with the brain and heart as the next focal points for the industry.
Intellia brought along all of the gene editing stocks including Beam Therapeutics (NASDAQ:BEAM), Editas Medicine (NASDAQ:EDIT), and CRISPR Therapeutics (NASDAQ:CRSP) just to name a few. Many of these are now trading at excessively high valuations as investors predict a surge of genomics related success stories moving forward. Investors should tread carefully after stocks have run up so high so fast, as there usually is a better entry point once the market becomes less frothy. For now, we should bask in the incredible accomplishment that Intellia has provided, and start doing some research into what could be the next big industry to invest in for the future.
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