Intellia Therapeutics is confident in the safety profile of its gene therapies after revealing that a man’s death in a Phase III trial was not related to liver failure.
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.
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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 Phase III trials, including the MAGNITUDE and the MAGNITUDE-2 trials (NCT06672237), both investigating nexiguran ziclumeran, and the Phase III HAELO trial (NCT06634420) of lonvoguran, also an in vivo CRISPR therapy, in hereditary angioedema (HAE).
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By GlobalDataWhile enrolment in both MAGNITUDE trials is currently under a clinical hold by the US Food and Drug Administration (FDA), enrolment has concluded in the HAELO trial. Leonard added that top line data from HAELO is due to be presented in mid-2026, with hopes to submit a biologics licence application (BLA) in the H2 with a commercial launch expected in H1 2027.
Speaking on safety signals in the MAGNITUDE trials, Leonard said: “If you look across the Phase III MAGNITUDE trial, the incidence of Grade 4 transaminases is less than 1% across that entire study. We’re working through the clinical hold with the FDA, and we’re actively engaged in that process with them.”
Intellia does have another candidate, REGV-131, developed in collaboration with Regeneron Pharmaceuticals, which is in a Phase II trial (NCT06379789) in haemophilia B.
While there have been some companies pulling out of the gene therapy space, there is still clear interest from big pharma, according to a GlobalData report.
GlobalData’s Strategic Intelligence report, ‘Cell and Gene Therapy Investment Trends‘, reveals that approximately 50% of cell and gene therapy (CGT) venture capital (VC) activity is focused at the Series B-stage, when companies usually shift from platform validation to clinical execution.
GlobalData’s senior healthcare researcher Irena Maragkou says: “Big pharma continues to demonstrate willingness to pay a premium for some late-stage or clinically validated assets when they align with long-term strategic objectives and portfolios.”
While oncology-focused CGT deals remain heavily skewed toward early R&D and gene-modified cell therapies, the merger and acquisition (M&A) activity centred on non-oncology CGT assets has become more mature and diversified.
Maragkou concludes: “As therapeutic CGT approvals increase, and the CGT market is expected to grow at a rate of 34.2% by 2031, companies must simultaneously prepare for sector-specific challenges such as regulatory complexity and manufacturing scalability. Therefore, biotech companies need to be strategic in investing in differentiated technologies and build execution capabilities to deliver clinical and commercial impact.”
GlobalData is the parent company of Clinical Trials Arena.
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