We can now edit genes inside the body. That sent this biotech’s stock soaring

Intellia is one of a slew of biotechs focused on CRISPR gene-editing, an almost sci-fi seeming medical advancement that could potentially be used to treat everything from cancer to inherited genetic disorders. To put it simply, CRISPR utilizes a slice-and-dice approach to fixing genomic abnormalities. Is there a particular genetic sequence within your cells that, say, creates a certain protein that leads to blindness? CRISPR can be used to selectively chop off those tiny bits of aberrant genomic code and then paste in the good stuff to address the disease.

While the technology is still fairly new, academic institutes and biopharma companies have been working on CRISPR-focused treatments for years, and the gene-editing platform has massive market potential across dozens of diseases (not to mention the ability to fix maladies on a fundamental biological level). Jennifer Doudna, the renowned biologist and UC Berkeley professor who is a co-founder of Intellia, was one of the winners of the 2020 Nobel Prize in Chemistry for her foundational CRISPR research.

The latest advance for CRISPR and Intellia has to do with how CRISPR can be utilized. Investors are pouncing on the stock because of a new study showing that CRISPR technology, for the first time ever, can be used in vivo. That’s a fancy way of saying “inside the body,” as opposed to in vitro or ex vivo. With ex vivo drug development, you would take biological material out of a body before deploying an experimental treatment and then re-injecting it into a patient.

But with an in vivo approach, you can send a CRISPR-based therapeutic straight to a certain part of the body and then let the cells in that particular organ take care of the rest of the genetic-modifying work. In the case of Intellia’s therapy, NTLA-2001, it uses a lipid (i.e., fat) nanoparticle in order to deliver the treatment to the liver. It’s a two-part process: First, use an RNA guide to target the problematic gene, and partner it with a messenger RNA (mRNA, or the kind of gene-encoding technology that’s at the heart of the multiple COVID vaccines) to initiate the actual genomic slice-and-dice.

In interim study results published in the New England Journal of Medicine, NTLA-2001 was found to reduce the level of a disease-associated protein in the liver called transthyretin by 87% with a high dose of the treatment. That’s a big improvement on current standard treatments, and a promising outcome for patients with transthyretin amyloidosis (ATTR), a rare degenerative disease that can lead to nerve damage and the stiffening of the heart.

“The data we presented this week demonstrate how important it is to tackle all of the challenges in clinical applications of CRISPR,” said John Leonard, M.D., Intellia Therapeutics’ president and CEO, in an emailed statement. “By developing complete solutions for both editing and delivery, we have created not just a minimum viable product but a versatile, modular platform that can be applied to a broad spectrum of diseases.”

Analysts were also excited about the news. “We think data presented today is a home run for [Intellia],” wrote RBC analyst Luca Issi in a research note upgrading the stock’s outlook, raising the price target from $110 to $150. Other analysts noted Intellia’s results could raise the prospects of gene-editing focused companies at large since it is, in essence, a proof of concept which now has data to back it up. We may soon see other firms such as Editas, Beam Therapeutics, and some larger life sciences companies rush into the breach.