Intellia's single-dose CRISPR therapy slashes hereditary angioedema attacks by 87% in Phase 3 trial

Intellia Therapeutics reported that its CRISPR-based gene editing drug lonvo-z achieved a dramatic reduction in swelling attacks for patients with hereditary angioedema (HAE) in a Phase 3 study.

The trial enrolled 80 participants, and those receiving the single dose saw an 87% drop in attack frequency compared with placebo. More than 60% of treated patients experienced no attacks at all during the observation period, versus only 11% in the control group.

The results position lonvo-z as a potential second CRISPR medicine to win regulatory approval, following Vertex’s Casgevy for sickle cell disease, and mark the first in‑vivo CRISPR therapy to reach this stage. Intellia has begun a rolling submission with the FDA.