Problem:
Preclinical data in animal models suggest that gene editing by NTLA-2002 is durable and that the edits are passed to daughter cells during cell division in the liver.14,15 NTLA-2002 therapy permanently alters hepatocyte DNA and thus has the potential for long-term control of hereditary angioedema after a single dose, unlike the treatments for hereditary angioedema that temporarily disrupt protein expression or function and are administered on a long-term basis. NTLA-2002 treatment was safe in this small study, but longer follow-up will be needed for a robust assessment of safety and efficacy outcomes in patients with hereditary angioedema. All the patients enrolled in this phase 1 study had varying numbers of angioedema attacks per month during the historical attack period. Some patients had more severe disease than other patients at baseline (Fig. 2), and the time needed to control the angioedema attacks was longer in these patients. Nine of the 10 patients remained free of angioedema attacks from the end of the primary observation period through the latest assessment. Need Assignment Help?