Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.
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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.
Excerpted from N Engl J Med
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- N Engl J MedPickLonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.
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.
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