Amid Safety Investigation, ATTR Gene Therapy Buoyed by Good 3-Year Data

The pause in Intellia's Phase III trials for nexiguran ziclumeran due to safety issues, despite positive 3-year data, highlights the delicate balance between efficacy and safety in gene therapy development. Biotech/pharma professionals will closely monitor how this event shapes regulatory scrutiny, patient safety considerations, and the future development of CRISPR-based therapies.