Intellia Therapeutics temporarily paused dosing and screening for its phase 3 Magnitude and Magnitude-2 trials of Nex-z following a patient hospitalization, according to an Oct. 27 news release.
The patient, who had been hospitalized with severe liver-related side effects, has since died, Intellia said in a Nov. 6 update.
The company reported Oct. 24 a case of grade 4 liver transaminases and elevated total bilirubin in a patient who received Nex-z on Sept. 30. The patient has been hospitalized and is receiving medical care.
Nex-z is a CRISPR-based gene therapy designed to inactivate the TTR gene in patients with transthyretin amyloidosis. More than 650 patients with cardiomyopathy are enrolled in Magnitude, and 47 patients with polyneuropathy are enrolled in Magnitude-2. Intellia estimates that more than 450 patients have received Nex-z to date.
The Cambridge, Mass.-based biotech company is working with global regulatory authorities and clinical experts to determine next steps, President and CEO John Leonard, MD, said.
Editor’s note: This article was updated Nov. 7 to reflect the patient’s death.
