By Technology Networks –
A single treatment with, a CRISPR-Cas9-based gene editing therapy, is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful and sudden onset of swelling, sometimes resulting in death. Confirming the findings published earlier this year by researchers from Amsterdam UMC, the University of Auckland and Cambridge University Hospitals NHS Foundation Trust. This phase two study is published today in the New England Journal of Medicine and presented at the American College of Allergy, Asthma & Immunology’s annual congress on the 26th of October.
“The results of this double-blind, placebo-controlled portion of the study confirm our promising findings from the phase 1 study, showing dramatic reductions of angioedema swellings following a single-dose treatment with this gene editing-based therapy,” says Danny Cohn, internist at Amsterdam UMC and first author of the study.
