Shares in US biotech Intellia Therapeutics (Nasdaq: NTLA) slid by a quarter on Thursday following a safety update from its late-stage trial of a CRISPR-based treatment for a rare heart disease.
The company disclosed that one patient in its Phase III study experienced elevated liver enzyme levels, raising concerns over the experimental therapy's risk profile.
The treatment, nexiguran ziclumeran, is being evaluated in the MAGNITUDE trial for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition caused by misfolded proteins that build up in the heart. Although the side effect was serious based on lab findings, the patient showed no symptoms, required no hospitalization, and enzyme levels have started to decline.
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