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Wolfgang Miesbach: One-Time CRISPR Therapy Reduces Hereditary Angioedema Attacks by 87%
Wolfgang Miesbach/LinkedIn
Jun 17, 2026, 04:59

Wolfgang Miesbach: One-Time CRISPR Therapy Reduces Hereditary Angioedema Attacks by 87%

Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn about a recent article by Danny M. Cohn et al, published in NEJM, adding:

”One-Time CRISPR Infusion Cuts HAE Attacks by 87%.

In the phase 3 HAELO trial, a single 50 mg IV infusion of lonvoguran ziclumeran (lonvo‑z) — an in vivo CRISPR-based therapy targeting KLKB1 — led to a profound and durable reduction of hereditary angioedema (HAE) attacks.

KLKB1 encodes plasma prekallikrein; by introducing precise edits in hepatocyte DNA, lonvo‑z durably reduces prekallikrein production in the liver, rebalancing the kallikrein–kinin pathway and lowering bradykinin-driven angioedema risk.

Dosing

  • One-time, 50 mg intravenous infusion (no repeat dosing through week 28)
  • Long-term prophylaxis was washed out and not permitted during weeks 5–28

Efficacy (weeks 5–28)

  • Mean monthly attack rate: 0.26 with lonvo‑z vs 2.10 with placebo (−87%; P<0.001)
  • 62% of patients on lonvo‑z were completely attack‑free without long-term prophylaxis (vs 11% with placebo)
  • On‑demand–treated and moderate/severe attacks were reduced by ~90%; AE‑QoL improved by −23.5 points, well above the minimal clinically important difference

Safety

AEs in 92% (lonvo‑z) vs 86% (placebo), all grade 1–2 in the lonvo‑z arm; no serious or grade ≥3 events reported with lonvo‑z.

Most frequent events: infusion‑related reactions (62%), headache, fatigue, back pain, upper respiratory tract infection; liver enzyme elevations were mild, transient, and self‑limiting.”

Title: Lonvoguran Ziclumeran — In Vivo CRISPR Gene Editing in Hereditary Angioedema

Authors: Danny M. Cohn, Padmalal Gurugama, Hilary J. Longhurst, Emel Aygören-Pürsün, Timothy J. Craig, Henriette Farkas, Joshua Jacobs, William R. Lumry, Markus Magerl, Jonny Peter, Marc A. Riedl, David Maag, Adele Golden, Mrinal Y. Shah, Andrea Sutherland, Catherine R. Miller, Ahmed M. Abdelhady, Yuanxin Xu, James S. Butler, David Lebwohl, John Leonard, Aleena Banerji

Wolfgang Miesbach: One-Time CRISPR Therapy Reduces Hereditary Angioedema Attacks by 87%

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Adele Golden Ahmed M. Abdelhady Aleena Banerji Andrea Sutherland Catherine R. Miller CRISPR Danny M. Cohn David Lebwohl David Maag Emel Aygören-Pürsün HAE HAELO Health Hematology Hemostasis Hemostasis News Hemostasis Today Henriette Farkas Hereditary Angioedema Hilary J. Longhurst James S. Butler John Leonard Jonny Peter Joshua Jacobs KLKB1 lonvoguran ziclumeran Marc A. Riedl Markus Magerl Medicine Mrinal Y. Shah Padmalal Gurugama Timothy J. Craig William R. Lumry Wolfgang Miesbach Yuanxin Xu
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