What Is CRISPR and Why Should Investors Care?

- September 16th, 2016

CRISPR-Cas9 is revolutionizing gene engineering. And for those keen on the sector, there is ample opportunity to break into this market, thanks to the rise of CRISPR-based biotechs like Editas Medicine or Intellia Therapeutics. But before you buy in, read on. This emerging technology has plenty of potential—but also a few pitfalls.

CRISPR: while it may sound like a favorite snack food, the term actually stands for Clustered Regularly Interspaced Short Palindromic Repeats. But what does that mean in plain English? The short answer: a revolution in gene engineering.
No wonder that CRISPR has piqued the interest of many life science investors. And for those keen on the sector, there is ample opportunity to break into this market, thanks to the rise of CRISPR-based biotechs like Editas Medicine (NASDAQ:EDIT) or Intellia Therapeutics (NASDAQ:NTLA).
But before you buy in, read on. This emerging technology has plenty of potential—but also a few pitfalls.

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What are CRISPRs?

Bacteria and other microorganisms have repetitive, somewhat palindromic sequences of DNA called ‘CRISPRs.’ Between these are ‘spacers’—or DNA copied from viruses that once attacked the bacterium. In this way, “spacers serve as a ‘genetic memory’ of previous infections,” Ekaterina Pak, a Biomedical PhD candidate explained on  Harvard’s Science in the News blog.
Spacers are created as follows:

  1. A new virus attacks. The bacterium processes the viral DNA, creating new spacers in its CRISPR sequence.
  1. The viral DNA is transcribed into RNA, a single strand molecule.
  1. Working as a guide, these RNA molecules instruct the bacterium’s molecular machinery to neutralize the invading virus. Should the same pathogen attack later, the cell already has instructions on how to eliminate it.

Genetic engineering

CRISPR immunity, as it’s called, is an interesting discovery—but on its own, not enough to capture so much scientific or investor attention. The revolutionary aspect of this finding has to do with its implications for genetic engineering.
Researchers can hijack the processes behind CRISPR immunity and instead use them for gene editing. They synthesize RNA molecules, mimicking the activity of the bacterium. These lab-made molecules then serve as a guide for cellular behavior: they direct the Cas9 enzyme to a specific DNA sequence, where it severs the strands in order for new genetic material to be inserted or taken away.
In this way, scientists can manipulate a single gene—and they have. CRISPR-Cas9, as the technology is known, has been used to genetically modify any number of organisms, ranging from a fruit fly to a human cell.
It’s not the first way scientists have found to edit a genome—but it is the best. CRISPR-Cas9 is faster and more cost-effective than previous methods. It’s also far more precise.
But that’s not to say it’s perfect—at least not yet. CRISPR-Cas9 does make inaccurate cuts at times, and there’s more work that needs to be done before it can be used in clinical therapies.

Medical applications

Unsurprisingly, CRISPR-Cas9 is expected to be key in treating genetic diseases going forward—in fact, scientists from MIT have already leveraged it to cure a liver disorder in mice. One day in the not so distant future, CRISPR-Cas9 may cure diseases like cystic fibrosis or sickle cell anemia.
CRISPR-Cas9 could also lead to a whole spate of improved antibiotics. Theoretically, the technology can help create drugs that only target bad bacteria, leaving the rest alone.

The controversy

There is a scary side too: critics warn that CRISPR-Cas9 may result in a ‘designer baby’ trend, with expectant parents controlling every aspect of their offspring’s genetics.
As such, the ethics behind using CRISPR-Cas9 on human embryos is a hot topic. Many scientists warn against this application, arguing that the effects of such intervention cannot be known until birth. They further suggest that it is immoral to make changes that will be inherited by generations to come.
In fact, the 2015 International Summit on Human Genome Editing concluded that genetically modified embryos or germline cells should not be used in insemination.
And the controversy doesn’t end there. Already, CRISPR-Cas9 has been used to make domesticated pigs immune to swine flu and respiratory viruses. But as Nature magazine noted, these genetically modified animals raise new questions: should they be regulated in the same way as other GMOs, since they don’t actually possess another species’ DNA?

What that means for investors

CRISPR-Cas9 has changed the landscape of genetics investing, making more targeted cures and treatments seem possible. Diseases previously labelled incurable might one day disappear. That creates new hope for patients, and new opportunity for investors.
Still, the legal and ethical implications of genetic engineering have yet to be determined. That makes it is challenging to predict how these future products might be regulated in years to come.
 
Don’t forget to follow us @INN_LifeScience for real-time news updates.
Securities Disclosure: I, Chelsea Pratt, hold no direct investment interest in any company mentioned in this article.

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