Yan Leyfman, Medical Oncologist, Co-Founder and Executive Director of MedNews Week, shared a post on LinkedIn:
“A single infusion. Durable gene editing. Remarkable reduction in hereditary angioedema attacks.
The phase 3 HAELO trial demonstrated that lonvoguran ziclumeran (lonvo-z), an investigational in vivo CRISPR gene-editing therapy, significantly reduced hereditary angioedema (HAE) attacks after a single intravenous infusion.
Key findings:
- 87% reduction in monthly HAE attack rate versus placebo (P<0.001)
- Monthly attack rate: 0.26 vs 2.10 attacks/month
- Single 50-mg intravenous infusion
- Median follow-up: 7.5 months
- No serious or grade ≥3 treatment-related adverse events reported
Hereditary angioedema is a rare, inherited disorder characterized by recurrent episodes of potentially life-threatening swelling. Current preventive therapies often require lifelong repeated dosing. In contrast, lonvo-z is designed as a one-time in vivo CRISPR gene-editing treatment, targeting the underlying biology of the disease.
If these results are confirmed with longer-term follow-up, this approach could fundamentally change the treatment paradigm—from chronic disease management to durable genetic intervention.
This study also represents another important milestone for in vivo CRISPR therapeutics, demonstrating that precision gene editing can translate into meaningful clinical benefit in a randomized phase 3 trial.”
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, and Aleena Banerji, the HAELO Investigators
Read the article here.
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