The FDA has halted clinical testing of an Intellia Therapeutics gene-editing therapy following a report that a patient in a Phase 3 study was hospitalized with liver complications.
Intellia said the liver problems met the trial protocol’s criteria for pausing the studies , which the company did on Monday. The therapy, nexiguran ziclumeran, or nex-z, is an experimental treatment for the rare disease transthyretin amyloidosis (ATTR). According to Intellia, the injured patient received nex-z on Sept. 30. On Oct. 24, this patient developed high levels of liver enzymes and compounds indicative of damage to the organ. Intellia said the FDA on Wednesday verbally notified the company that the two Phase 3 tests of the experimental therapy are now under a clinical hold.
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