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JPM26: Intellia Therapeutics hopeful on CRISPR safety despite patient death [Yahoo! Finance]
Last regeneron pharmaceuticals, inc. earnings: 2/6 06:30 am
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US:NASDAQ Investor Relations: investor.regeneron.com
In November 2025, Intellia announced that in the MAGNITUDE trial (NCT06128629) of nexiguran ziclumeran, an in vivo CRISPR therapy, for transthyretin (ATTR) amyloidosis, a male patient in his 80s died after receiving the study drug. The therapy is being developed in partnership with Regeneron. Speaking at the JP Morgan Healthcare Conference 2026, Intellia CEO John Leonard said that the patient attended the emergency room due to abdominal pain, where tests revealed elevated transaminase. During his stay in the hospital, it was revealed that the patient had air in his abdomen and received surgery where a perforation and ulcer were found. The patient died following a case of sepsis. The update on the circumstances of the patient's death may provide investors with more confidence in the safety of the gene therapy, especially given liver-associated patient deaths in gene therapy studies by other companies, including Sarepta and Pfizer Leonard said that the company has three ongoing
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