Intellia Therapeutics Inc (NASDAQ: NTLA) said on Saturday that her clinical trial, which used Crispr technology to treat a genetic disorder in the human body, showed positive results. The biotech company had partnered with Regeneron Pharmaceuticals Inc (NASDAQ: REGN) to the study using Nobel Prize-winning technology.
Intellia shares rose about 55% Monday morning. The stock opened at $135 per share at the start of the year, versus a much lower $56 per share. At the time of writing, the Cambridge-headquartered company is valued at $9.01 billion.
Intellia’s results mark a breakthrough for genetic studies
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The hereditary disease called transthyretin amyloidosis, which is under investigation, is characterized by abnormally high levels of a harmful liver protein that deposits in organs and tissues. The results showed that the Crispr technology helped treat the disease as it reduced levels of the harmful protein. It was the first clinical trial to use Crispr technology to treat a genetic disorder.
The early-stage trial marks a breakthrough for genetic studies as it fuels hopes that precise editing of human DNA (CRISPR technology) will one day help cure genetic disorders. Intellia Therapeutics CEO John Leonard said ahead of the results:
“It feels like we are walking through a door to all kinds of new possibilities here. And there aren’t many times in medicine where you can experience that.”
Intellia’s NTLA-2001 therapy showed no adverse effects
The results of the Intellia/Regeneron study were published this weekend in the New England Journal of Medicine. Eric Topol, geneticist and director of the Scripps Research Translational Institute, also expressed excitement about the future potential of Crispr therapies in the treatment of genetic disorders. He said:
“We are moving much faster with Crispr than anyone thought.”
Intellia/Regeneron call their therapy NTLA-2001. It’s a single-dose treatment that took just weeks to reduce levels of a harmful protein by about 96%, according to clinical trial results. No side effects were reported during the first four weeks of the study.
Regeneron Pharmaceuticals is now trading hands for $558 a share after recovering from a low of $446 a share in the first week of March. In terms of market capitalization, Regeneron is a much larger company than Intellia, with a valuation of over $59 billion and a price-to-earnings ratio of 15.79.
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