- Intellia Therapeutics soared 63% on Monday after it released promising data from its Phase I trial of a gene-editing CRISPR drug.
- Intellia said it was successfully able to genetically edit cells inside the liver of several patients.
- The phase I trial represents the first-ever in human data of CRISPR gene-editing technology.
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Shares of Intellia Therapeutics soared as much as 63% early on Mondayafter the company released promising data from its ongoing phase one trial of a gene-editing CRISPR drug.
The trial represents the first-ever in human clinical data of in vivo CRISPR genome editing, which showed no serious adverse events and an encouraging safety profile, according to the company.
The Phase one trial of NTLA-2001, which had a total of six patients enrolled, targeted a treatment for transthyretin amyloidosis. NTLA-2001 was designed to inactivate the TRR gene in liver cells to prevent the production of misfolded transthyretin protein (TTR), which accumulates in tissues and causes often fatal complications.
Interim data from the trial, which was published in the New England Journal of Medicine over the weekend, showed that a single 0.3mg/kg dose of NTLA-2001 led to an 87% mean reduction in serum TTR, with a maximum 96% serum TTR reduction by day 28.
"This is exciting early data both for people living with this devastating disease and for the entire scientific community working to maximize the potential of genetics-based medicines through cutting-edge research and technologies," said George Yancopoulos, the president of Regeneron.
Intellia partnered with Regeneron in 2016 to advance the CRISPR technology for in vivo therapeutic development. Shares of Regeneron were up 3% in early Monday trades.
Intellia now expects to begin an expanded single-dose phase one trial and will present additional data from the study later this year.
Following the data, Goldman Sachs, which rates Intellia as a "Buy," raised its price target on the company to $163, suggesting potential upside of 20% from current price levels. Goldman called Intellia's data "impressive" and said that it "unlocks a new era of one-time curative medicine."