18 articles · Updated · The Wall Street Journal · Apr 27
A single infusion of Intellia's therapy cut hereditary angioedema swelling attacks by 87%, with over 60% of treated patients attack-free for six months, compared to 11% on placebo.
The late-stage trial results prompted Intellia to begin seeking FDA approval, aiming for a U.S. launch in the first half of 2027. The therapy offers a one-time treatment by permanently inactivating the KLKB1 gene.
Current hereditary angioedema treatments require frequent dosing and do not always prevent attacks. Intellia’s shares had previously declined after a separate trial was halted due to a patient death and FDA hold.
Beyond rare disorders, could this in-body gene editing technology one day tackle more common conditions?
Will this one-time treatment model, offering a potential cure, disrupt the entire pharmaceutical industry?
As gene editing 'cures' become reality, how will healthcare systems handle their multi-million dollar price tags?
With DNA permanently altered, what are the lifelong health risks, and how will patients be monitored?
How can we be sure editing one gene won't accidentally break another, causing future health problems?