By Matt McCallFeb 01, 2019
The future of medicine is all about the personalization of medicine. That means that doctors will be able to tailor treatments that fit your specific ailments. Not the general ailments of a large population of people.
Today, medicine is designed to treat the most common symptoms of a disease. So everyone with that same disease will be given the exact same treatment. But considering we all have different genetic makeups, this method is clearly not ideal.
That is where gene editing – the ability to edit a human’s genome – comes into play.
The term “gene editing” oftentimes has a negative connotation. And given some of the post-apocalyptic movies that have become popular over the years, I can understand why some people are afraid. But genetic editing has so much to offer to the future of medicine.
A couple months ago, a Chinese scientist performed the first gene edit on a human by removing the gene that causes HIV in several newborns. The scientific community was not happy as most believe the science it not far enough along to verify its safety. However, the scientist proved that gene editing does have a place in the healthcare of tomorrow.
There are currently more than 10,000 known monogenic diseases that do not have a cure. A monogenic disease is caused by the mutation of one gene. Yes, one single gene. Imagine being able to repair that gene and cure a disease immediately. That’s exactly what science is working toward.
Sickle cell disease is one of the terrible ailments that falls into this category. Today, those with this disease live with ongoing pain and the knowledge that their life will likely be shortened. But soon, science will be able to cure these people with a single procedure.
Imagine the diseases that could be irradiated through the practice of gene editing. There are many companies working toward this goal, and today I’d like to introduce you to three of the major players in the space.
CRISPR is the largest of the Big Three gene editing companies, and it has a slight lead in the race. The company made headlines in October when the FDA removed its clinical hold on CTX001, CRISPR’s gene therapy to treat sickle cell disease.
Then, on January 4, the FDA granted the therapy Fast Track status. This is extremely important because it allows for more frequent interaction with the FDA review team as well as a rolling review of the marketing application.
CRISPR Therapeutics is backed by some powerful pharma partners. Combine that with the promising treatments making headway in the United States and Europe and it is clear that this company is a great place to start investing in this innovative and inspiring trend that should make investors a lot of money.
Editas falls in the middle of the Big Three gene editing companies. It can trace its roots back to the biggest players in the industry, specifically Feng Zhang, whose affiliation with the Broad Institute has given Editas a significant leg up.
The Broad Institute owns the patents to use CRISPR technology in eukaryotic cells – more complex cells that have a nucleus where the genetic material is found – so it puts Editas in an enviable position since it has the exclusive rights to the patents for any genetic target it chooses.
This company’s promising pipeline and connections make me confident that it is a stock investors must own to profit from gene editing over the long term.
This company doesn’t get the press of its fellow gene editing leaders. It is also the smallest of the three, but its upside is just as huge as the others.
I say that because Intellia stands out thanks to its proprietary lipid nanoparticle delivery (LNP) technology. This takes drug therapies directly to the genes that need to be edited and does it right there rather than having to extract the gene edit it in the lab. The process has proven to be safer and more effective than the alternatives.
Intellia has many irons in the fire as well as collaborations with two successful pharmaceutical companies, and I suspect it could have several therapies in FDA trials in the coming years. The key for gene editing companies will be to continue their partnerships and show progress, and that’s exactly what Intellia is doing.
I want to make one final note… gene editing stocks are what I call buried treasure stocks. That means that the big returns I believe are possible take patience and time. Most investors don’t have the long-term outlook that this kind of world-changing technology requires.
If that’s you, here is my suggestion: Go out and dig a big hole in your backyard. Bury these stocks in it, and in 10 years you can dig up your buried treasure. Metaphorically speaking, of course.
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