A look at a biotech company that represents a solid trend in the industry.
While the world is currently eyeing bitcoin as a way of getting rich overnight—or losing your shirts—Keith Speights, writing for The Motley Fool, takes a look at a biotech company that represents a solid trend in the industry. It’s worth noting, of course, that biotech investments are notoriously volatile, although compared to bitcoin and other cryptocurrency, biotech investment seems positively staid and middle-of-the-road.
The technology in question is gene editing, specifically CRISPR-Cas9, and the company Speights is focused on is Editas Medicine.
Gene editing is pretty much what it sounds like—cutting, deleting, inserting or replacing pieces of DNA. The technology has been around since at least 1991, but it was slow and expensive. In 2012, CRISPR, which stands for clustered regularly interspaced short palindromic repeats, used a bacterial enzyme called Cas9 to easily alter DNA.
There are a number of other companies using CRISPR, including CRISPR Therapeutics, Intellia Therapeutics, Juno Therapeutics, Vertex Pharmaceuticals and others.
“What sets Editas apart from the pack?” writes Speights. “Patents. In 2014, the company licensed patents for CRISPR-Cas9 held by the Broad Institute and Harvard University. These patents cover any use of the gene-editing technology in humans. Unless the patents are somehow invalidated (and they’ve been upheld once in court already), any company that develops a gene therapy using CRISPR-Cas9 will have to pay royalties to Editas.”
None of these companies actually have products on the market that have been developed with CRISPR, although they’re working on it. Editas is developing gene therapies for eye diseases as well as for cystic fibrosis (CF), Duchenne muscular dystrophy (DMD) and sickle cell disease. With Juno, it’s working on cancer therapies.
Although most scientists recognize the revolutionary benefits to CRISPR, many also feel that the industry is sort of glossing over the potential problems with the technology. It boils down to potential issues with what are dubbed downstream alterations. That is to say, part of what CRISPR does is cut the DNA at a specific, programmed section of DNA. But with 2 billion or so base pairs in the human genome, there are plenty of opportunities for CRISPR to make other unwanted and unintended cuts in the DNA. There was a groundbreaking but also ethically controversial study where researchers claimed to safely correct mutations in human embryos using CRISPR.
Michael Le Page, writing for New Scientist in September 2017, noted, “The first studies to try using the CRISPR genome-editing technique to alter the DNA of human embryos revealed several major problems. For instance, it only corrected mutations in a small proportion of embryos. Last month, however, a team led by Shoukhrat Mitalipov of Oregon Health and Science University claimed they had managed to improve efficiency while avoiding other key problems such as unwanted alternations. The study was widely proclaimed as a breakthrough. Now doubts have been raised. Genome editing works by breaking DNA, and letting a cell’s natural repair mechanisms fix it. This is usually quite haphazard, and precise repairs were thought to be rare.”
Which is a nice way of saying the technology may not be quite ready for prime time. Which is something Speights recognizes. “The process for getting rich with these three stocks is pretty simple. It involves only two steps. First, buy the stocks. Second, hold on to them for a long time. The first step is easy, but the second step could be really tough to do. All these stocks [Micron Technology, Alibaba and Editas] could be pretty volatile. That’s especially true for Editas Medicine, since it isn’t profitable yet and could have to raise cash through dilution-causing stock offerings. That volatility could make it difficult to stay calm at times,” Speights writes.
