FDA accepts the lonvo-z BLA with priority review and sets a decision date of 10 March 2027

Intellia Therapeutics/Lonvo-z (NTLA-2002)

The FDA accepted Intellia's application to market lonvo-z, a one-time CRISPR treatment for hereditary angioedema (HAE), a genetic disease that causes sudden, sometimes life-threatening swelling. Priority review sets a decision date of 10 March 2027, and the FDA does not currently plan an advisory committee. If approved, it would be the first CRISPR therapy edited inside the body to reach the market.

• Mechanism: one infusion permanently disables the KLKB1 gene in liver cells, cutting plasma kallikrein, the enzyme that drives the swelling attacks.
• Phase 3 HAELO (80 patients aged 16+, single 50 mg dose): 87% fewer monthly attacks than placebo over weeks 5–28; 62% were attack-free and off all HAE therapy, versus 11% on placebo.
• Side effects were mild or moderate (infusion reactions, headache, fatigue); no serious adverse events in the lonvo-z arm.
• The trade-off: today's HAE drugs are taken for life but can be stopped; a gene edit cannot be undone.