Shares of Intellia Therapeutics (NTLA) are climbing Monday after the company unveiled fresh Phase 3 data for its experimental CRISPR-based therapy for hereditary angioedema (HAE), a rare genetic disorder that causes sudden, painful swelling attacks.
The results, presented over the weekend at the European Academy of Allergy & Clinical Immunology Annual Congress and published in the New England Journal of Medicine, build on earlier findings that the study met its primary goal. During the efficacy evaluation period from weeks five through 28, patients who received lonvo-z (formerly known as NTLA-2002) saw an 87% reduction in monthly HAE attacks compared to those on placebo.
Even more striking: 62% of patients in the lonvo-z group remained completely attack-free and therapy-free over the six-month period, versus just 11% in the placebo arm. That difference hit a key secondary endpoint with statistical significance.
Intellia also reported that lonvo-z showed a favorable safety and tolerability profile. All treatment-related side effects during the primary observation period were mild or moderate, and no serious adverse events occurred among patients receiving the therapy.
Lonvo-z is designed as a one-time, in vivo CRISPR gene-editing treatment. It works by inactivating the KLKB1 gene after a single dose, aiming to permanently reduce kallikrein levels and prevent attacks. That's a different approach from chronic therapies that require regular injections.
William Blair analyst Myles Minter noted in a Monday research note that the additional data "further substantiated lonvo-z's competitiveness with Q4W donidalorsen on placebo-adjusted HAE attack rate reductions across multiple secondary endpoints." Donidalorsen, developed by Ionis Pharmaceuticals (IONS), was recently approved by the FDA as DAWNZERA for prophylaxis in patients 12 and older.
Minter added that the results strengthen Intellia's case for regulatory approval as the company prepares to complete a rolling Biologics License Application for lonvo-z in the second half of 2026.
Intellia shares were up 10.57% at $13.39 in Monday afternoon trading.













