Intellia Unveils Striking Long-Term Data on CRISPR Therapy Lonvo-z for Hereditary Angioedema at AAAAI 2026

Intellia Therapeutics, Inc., a pioneer in CRISPR-based gene editing, presented four compelling posters on its investigational therapy lonvoguran ziclumeran (lonvo-z; NTLA-2002) at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in Philadelphia.

The posters, now available on Intellia’s Scientific Publications & Presentations page, highlight deep, durable reductions in HAE attacks and kallikrein levels from a single 50 mg dose potentially ushering in a one-time treatment paradigm for this debilitating condition.

In the standout Phase 1/2 pooled analysis (n=32) titled “Long-Term Durability and Safety of Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) 50 mg in Patients with Hereditary Angioedema” (presented by Markus Magerl, M.D., Charité – Universitätsmedizin Berlin), patients achieved mean monthly attack rates of ≤0.2—a 96% reduction from baseline, over up to three years of follow-up. Notably, 97% (31/32) remained both attack-free and free of long-term prophylaxis (LTP), with attack-free periods spanning 2 months to 3 years.

A related poster, “Evolving Treatment Goals to Achieve Freedom from Attacks and Long-Term Prophylaxis Following a One-Time Treatment with Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002)” (presented by Aleena Banerji, M.D., Massachusetts General Hospital), reinforced these findings. Among 28 patients with >6 months of follow-up, 86% were attack-free and LTP-free, aligning with expert and patient priorities for minimizing treatment burden.

Supporting mechanistic insights came from “Quantitative Systems Biology Modeling Estimates Extent of Excessive Kallikrein Generation in Hereditary Angioedema Patients” (presented by Allen Kaplan, M.D., Medical University of South Carolina). The model demonstrated that HAE patients with C1-esterase inhibitor deficiency produce excess plasma kallikrein, driving bradykinin surges.

An 85% reduction in prekallikrein mirroring lonvo-z’s clinical effects normalized these levels, validating the therapy’s targeted gene inactivation of the KLKB1 gene.

Patient-centric challenges were addressed in “Chronic Medications Pose Challenges for People Living with Hereditary Angioedema” (presented by Paula Busse, M.D., Mount Sinai Hospital). Surveying 100 U.S. HAE patients (89% on LTP), it revealed 34% experienced ≥1 attack monthly, with only 20% attack-free in the prior year. Respondents prioritized eliminating chronic medications and boosting efficacy goals lonvo-z appears poised to meet.

Lonvo-z, leveraging Nobel Prize-winning CRISPR/Cas9 for in vivo gene editing, is advancing in the Phase 3 HAELO trial.

It holds FDA Orphan Drug and RMAT designations, U.K. MHRA Innovation Passport, EMA PRIME, and EU Orphan Drug status, signaling regulatory momentum for a potential first-in-class one-time HAE therapy.

These AAAAI data underscore lonvo-z’s transformative potential, offering sustained attack prevention without ongoing prophylaxis and addressing core unmet needs in HAE management.

 Reference

Intellia Therapeutics Presents Longer-Term Clinical Data for Lonvoguran Ziclumeran (lonvo-z); Hereditary Angioedema (HAE) Patient-Focused Research at AAAAI 2026, 03 March 2026, Intellia Therapeutics Presents Longer-Term Clinical Data for Lonvoguran Ziclumeran (lonvo-z); Hereditary Angioedema (HAE) Patient-Focused Research at AAAAI 2026 – Intellia Therapeutics

Scientific Publications & Presentations, Scientific Publications & Presentations – Intellia Therapeutics