Intellia Therapeutics has announced a pause in enrollment and dosing for two clinical trials of its CRISPR therapy, nex-z, aimed at treating transthyretin amyloidosis (ATTR), due to a serious adverse event involving liver toxicity that resulted in the hospitalization of a patient. This decision, communicated during a call with investors, underscores the potential risks associated with novel gene-editing therapies.
The pause comes at a critical time for Intellia, as the company seeks to establish the safety and efficacy of its CRISPR technology. The incident raises significant concerns regarding the long-term safety profile of gene-editing approaches in clinical settings, particularly in patients with pre-existing conditions. Regulatory bodies will likely scrutinize these trials closely, which could lead to delays in development timelines and impact investor confidence.
As the industry continues to navigate the complexities of gene therapy, this development serves as a reminder of the inherent challenges in balancing innovation with patient safety. Stakeholders across regulatory, quality assurance, and clinical development sectors will need to closely monitor the situation as Intellia works to address these safety concerns and resume its clinical programs.
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