Can Intellia's lonvo-z Turn CRISPR Promise into HAE Practice?

Intellia's lonvo-z (formerly NTLA-2002) is an investigational in vivo CRISPR gene editing therapy targeting the KLKB1 gene to permanently lower kallikrein and bradykinin levels in hereditary angioedema (HAE).

In a Phase 3 HAELO trial, a single dose of lonvo-z reduced HAE attack rates by 87% vs placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm versus 2.10 in the placebo arm.

Approximately 62% of lonvo-z patients were both attack‑free and therapy‑free over the six‑month evaluation period, compared with 11% in the placebo group.

Up to three years of follow‑up in Phase 1/2 data show a mean 98% reduction in monthly HAE attack rate and 100% of treated patients remaining attack‑free, with no serious adverse events reported after the first month.

Intellia has initiated a rolling Biologics License Application (BLA) submission to the FDA and is preparing for a potential U.S. launch of lonvo-z in the first half of 2027, making it the first in vivo CRISPR therapy and potentially the first one‑time treatment for HAE.