N Engl J Med·· 10 hours agoPickScore89
Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.
Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.
In brief
A single intravenous infusion of the in vivo CRISPR gene-editing therapy lonvoguran ziclumeran cut monthly hereditary angioedema attack rates by 87% relative to placebo in a phase 3 trial of 80 patients with C1 inhibitor deficiency. From weeks 5 through 28, the mean monthly attack rate was 0.26 with lonvo-z versus 2.10 with placebo (P<0.001), with no serious or grade 3 or higher adverse events in the treatment group. The results support a one-time infusion replacing ongoing preventive therapy for this disease.
Why it matters
Phase 3 evidence that one-time in vivo CRISPR editing nearly eliminates attacks in hereditary angioedema.
Source: N Engl J Med · doi.org