Intellia’s In Vivo CRISPR Therapy Hits Primary Goal in Phase 3 Trial for Hereditary Angioedema
Key Results
- Intellia Therapeutics announced that its CRISPR-based treatment, lonvoguran ziclumeran, met its primary endpoint in a Phase 3 trial for hereditary angioedema (HAE).
- The one-time treatment reduced attacks by 87% compared to placebo.
- Six months after treatment, 62% of patients were attack-free and not using other therapies.
Safety
- The company described safety and tolerability as "favorable."
- The most common side effects were infusion-related reactions, headaches, and fatigue.
- One patient in a separate Intellia trial died from liver toxicity, but no similar events were reported in this trial.
Significance
"First Phase 3 data for an in vivo CRISPR therapy targeting a disease-causing gene." – CEO John Leonard
- Unlike Vertex's Casgevy, which edits cells outside the body, Intellia's therapy is administered intravenously and edits cells inside the body.
Regulatory Path
- Intellia has started a rolling application with the U.S. FDA and plans to complete the filing in the second half of 2025.
- If approved, the company expects to launch the treatment in the U.S. in the first half of 2026.
Market Context
- The therapy would compete with approximately a dozen chronic HAE drugs.
- Genetic medicines have faced commercial challenges; for example, BioMarin withdrew its hemophilia A gene therapy due to weak sales.
- CEO John Leonard cited durability of effect as a differentiating factor, stating no cases of waning effect have been observed in nearly six years of follow-up.