Intellia Therapeutics (NTLA) shares surged 55.4% in the premarket after the Massachusetts-based company and partner Regeneron (REGN) announced positive results in a phase 1 study of a gene-editing treatment for a disease called transthyretin amyloidosis. Regeneron shares gained 1.6%. Two other companies involved with the same gene-editing technology also rallied in premarket trading, with CRISPR Therapeutics (CRSP) soaring 13.5% and Editas Medicine (EDIT) jumping 17.1%.
The data showed that for the six people that received a single 0.3 mg/kg dose of NTLA-2001, there was an 87% mean reduction in serum TTR, while there was a maximum 96% serum TTR reduction following a month’s treatment.
NTLA-2001 is intended to shut down the TTR gene in liver cells to stop the production of misfolded TTR protein, which can gather in body tissues and cause fatal complications of ATTR amyloidosis.
The treatment’s achievement is unique; the companies say the interim results amount to the first-ever clinical data showing in-vivo CRISPR genome editing in humans to be safe and effective.