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Intellia Therapeutics said on Monday its experimental gene-editing therapy reduced the frequency of swelling attacks in patients with a rare genetic disorder in a late-stage trial, sending its shares up more than 5% in volatile premarket trading.
The therapy lonvoguran ziclumeran is aimed at treating hereditary angioedema (HAE), an inherited disorder that causes recurring episodes of swelling in the limbs, face, gastrointestinal tract and airways, with symptoms including abdominal pain, nausea and vomiting.
In a trial involving 80 patients, a one-time infusion of the therapy reduced swelling episodes by 87% compared to placebo over six months, meeting the main goal.
The therapy also helped 62% of patients remain attack-free without regular preventive therapy during that period, compared with 11% in the placebo group, meeting the secondary goal of the trial.
The company’s shares surged 30% in premarket in anticipation of positive trial data before paring most of those gains.