FDA Places Clinical Hold on Intellia's Late-Stage CRISPR Trials After Severe Adverse Event

Intellia Therapeutics, a gene-editing company developing CRISPR-based therapies, is facing a significant setback as the U.S. FDA has placed a clinical hold on its two late-stage Phase 3 trials of nexiguran ziclumeran (nex-z), a treatment for transthyretin amyloidosis. This hold follows a serious liver-related adverse event in a trial participant, which led Intellia to voluntarily pause dosing and patient enrollment. The FDA's clinical hold introduces additional regulatory scrutiny and delays the trials, potentially postponing study readouts by several quarters. The company’s stock dropped approximately 14% following the announcement, reflecting investor concerns about the impact on Intellia’s development timeline and financial outlook. Despite the challenges, Intellia continues to explore gene editing therapies for various genetically defined diseases and maintains strong institutional ownership and insider buying. Analysts have downgraded the stock to a hold rating with revised price targets reflecting the increased uncertainty around the clinical program.