The Food and Drug Administration cleared Intellia Therapeutics to begin testing its CRISPR gene-editing technology in people on Thursday, leading NTLA stock to jump.
This is a first for a so-called in-vivo gene-editing drug. Intellia plans to use it to prevent swelling attacks in people with a genetic condition called hereditary angioedema.
With this approach, the genetic edit takes place inside the body. The treatment is also systemic, meaning a doctor infuses it into a patient's arm. From there, the gene-editing instructions must find their way to the proper organ. In this case, that's the liver.
While Intellia has tested its in-vivo technology in people in other countries, this is the first time the FDA has signed off on human testing. Before this, the agency has only allowed testing in people of ex-vivo gene editing — where researchers edit cells outside the body and then infuse them into patients.
"This is an important milestone for Intellia as it is the first-ever (investigational new drug application) cleared by the FDA for in-vivo gene editing," RBC Capital Markets analyst Luca Issi said in a report.
On today's stock market, NTLA stock surged 8% to close at 43.99. The move helped shares make a definitive move above their 50-day moving average, according to MarketSmith.com. NTLA stock has hovered just north of that key line since mid-February.
NTLA Stock: A Boon For CRISPR Companies?
Meanwhile, there are other treatments for hereditary angioedema, or HAE. Among those is a drug from Takeda Pharmaceutical called Takhzyro.
Intellia says its treatment led to a 65%-92% decrease in the level of a problematic protein tied to HAE. Further, Takeda's drug leads to a 60% decrease.
The move Thursday "signals that the FDA is willing to clear (investigational new drug applications) for indications where there are effective alternatives like HAE," Issa said. "We think this is a positive as it lifts an important overhang for the stock and the broader field of gene editing."
He kept his outperform rating and 120 price target on NTLA stock.
Another FDA Filing On Deck
Intellia noted on its recent earnings call that it plans to submit an investigational new drug application for its other in-vivo gene-editing drug in the middle of this year. That treatment would help tamp down on an abnormal protein that builds up on the heart.
The company is currently testing that drug in patients in France, New Zealand, Sweden and the U.K., according to ClinicalTrials.gov. Likewise, the hereditary angioedema drug has already been tested in people in the Netherlands, New Zealand and the U.K.
"The clearance today from U.S. regulators is another important milestone that highlights how Intellia continues to pave the way as the leading genome editing company," Intellia spokeswoman Rebecca Spalding said in an email to Investor's Business Daily.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
