https://www.clinicaltrialsarena.com/comment/crispr-ntla-2001-heart-disease/

 

The therapy previously made waves in the gene editing field in June 2021 with the release of positive interim data for the neuropathy arm of the trial. Although this data only provided analyses over a 28-day period, the publication of the first clinical data supporting the safety and efficacy of an in vivo CRISPR therapy was hailed as a watershed moment in genomics, culminating in soaring stock shares for Intellia. However, the latest data, which was obtained from the cardiomyopathy arm of the study, shows that NTLA-2001 induces sustained reductions in serum TTR, with patient follow-up ranging from four to six months. Critically, the durability of this response provides credence to Intellia’s objective of permanently inactivating the TTR gene, potentially curing the disease.