Intellia reports first clinical data for NTLA-2002 (lonvo-z) in hereditary angioedema

Intellia Therapeutics/Lonvo-z (NTLA-2002)

Intellia reported the first human data for NTLA-2002, a one-time CRISPR infusion that switches off the liver gene making kallikrein in hereditary angioedema. In September 2022 kallikrein fell 65% at 25 mg and 92% at 75 mg; by November the first patients had been attack-free for 5.5 to 10.6 months.

• NTLA-2002 targets KLKB1 gene (plasma kallikrein); single-dose CRISPR therapy, not chronic dosing.
• Dose-response: 25 mg = 65% kallikrein reduction; 75 mg = 92% reduction at week 8.
• Attack-free periods 5.5–10.6 months post-infusion (data through November 2022); sustained benefit observed.
• Phase 1 dose escalation, n=10 adult HAE patients; Phase 2 expansion planned for H1 2023.