NTLA · Intellia Therapeutics, Inc.

This 8-K discloses transformative clinical and regulatory progress for Intellia’s lead asset, lonvo-z, the first in vivo CRISPR therapy to report positive Phase 3 data. The HAELO trial met its primary endpoint with an 87% relative reduction in monthly HAE attacks — a statistically robust outcome (p<0.0001) that exceeds historical benchmarks for chronic HAE therapies. Critically, the durability signal is reinforced by 100% LTP-free status among lonvo-z recipients through median follow-up of 7.5 months, including those entering via blinded crossover after week 28. The safety profile — zero serious adverse events, exclusively Grade 1/2 events (mostly transient infusion reactions), and no concerning hepatic or hematologic signals — substantially de-risks near-term regulatory review. By initiating a rolling BLA, Intellia has moved decisively toward commercialization, aligning with its stated timeline for FDA filing completion in H2 2026 and potential launch in H1 2027. As highlighted in Exhibit 99.1, management frames these results as a 'global first for in vivo gene editing' and a 'paradigm shift' for HAE patients burdened by lifelong prophylaxis and unpredictable attacks. Given HAE’s rarity (~7,000 diagnosed U.S. patients) and high unmet need — underscored by cumulative $19.1B U.S. healthcare costs for chronic therapies — lonvo-z’s one-time, outpatient administration model represents a structurally differentiated value proposition with significant pricing and access implications. The absence of financial or operational disclosures means materiality derives entirely from clinical validation, regulatory momentum, and first-to-market positioning in a genetically defined, high-burden rare disease.