Intellia Therapeutics Inc (NASDAQ: NTLA) said on Saturday its clinical trial that used Crispr technology to treat a genetic disorder inside the human body showed positive results. The biotech company had collaborated with Regeneron Pharmaceuticals Inc (NASDAQ: REGN) on the study that used the Nobel Prize-winning technology.
Intellia shares were up about 55% on Monday morning. The stock opened at $135 per share versus a much lower $56 per share at the start of the year. At the time of writing, the Cambridge-headquartered company is valued at $9.01 billion.
Intellia’s results mark a breakthrough in genetic studies
The hereditary disease called Transthyretin amyloidosis under investigation is characterised by abnormally high levels of a harmful liver protein that deposits in organs and tissues. The results showed that Crisper technology helped treat the disease as it reduced the levels of the damaging protein. It was the first clinical trial that used Crispr technology to treat a genetic disorder.
The early-stage trial marks a breakthrough in genetic studies as it fuels hopes that the precise editing of human DNA (Crispr technology) will some day help cure genetic disorders. CEO John Leonard of Intellia Therapeutics said ahead of the results:
“There’s a feeling like we’re walking through a door here into all kinds of new possibilities. And there’s not many moments in medicine where you get to experience that.”
Intellia’s NTLA-2001 therapy didn’t show any adverse effects
The results of the Intellia/Regeneron study were published over the weekend in the New England Journal of Medicine. Eric Topol, geneticist and director of Scripps Research Translational Institute, also expressed excitement in the future potential of Crispr therapies in treating genetic disorders. He said:
“We’re moving far faster with Crispr than anyone thought we would.”
Intellia/Regeneron call their therapy NTLA-2001. It is a single-dose treatment that took only weeks to reduce the levels of a harmful protein by about 96%, as per the results of the clinical trial. No adverse effects were reported during the first four weeks of the study.
Regeneron Pharmaceuticals is now exchanging hands at $558 per share after recovering from a low of $446 per share in the first week of March. In terms of market cap, Regeneron is a much bigger company than Intellia, boasting a valuation of more than $59 billion and a price to earnings ratio of 15.79.
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