Objective:

To evaluate the efficacy and safety of a one-time in vivo CRISPR therapy (lonvoguran ziclumeran) in patients with hereditary angioedema due to C1 inhibitor deficiency.

Approach:

Key Findings:

• The lonvo-z group had a monthly attack rate of 0.26 compared to 2.10 in the placebo group, representing an 87% relative reduction.
• Attacks requiring on-demand treatment were reduced by 89% in the lonvo-z group versus placebo.
• Moderate-to-severe attacks fell by 91% in the lonvo-z group.
• 62% of patients receiving lonvo-z were attack-free between weeks 5 and 28 without long-term prophylaxis, compared to 11% in the placebo group.
• Patients in the lonvo-z arm showed greater improvement on an angioedema-specific quality-of-life measure.

Interpretation:

Limitations:

• The trial is limited by sample size and follow-up duration, with a median follow-up of 7.5 months.
• Longer-term monitoring is needed to assess durability and safety, including risks outside the controlled clinical trial setting.

Conclusion:

Sources:

• Press Release
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