Intellia Therapeutics (NTLA) Corporate presentation summary

Vision and strategy

• Aims to transform patient lives by treating root causes of disease using gene editing technology, offering single-dose treatments with potential lifelong benefits and reduced healthcare burden.

• Focuses on developing in vivo CRISPR-based therapies for severe diseases, with over 600 patients dosed and multiple publications in top medical journals.

Pipeline and market opportunities

• Two late-stage assets: Lonvo-z for hereditary angioedema (HAE) and Nex-z for transthyretin amyloidosis (ATTR), both with blockbuster potential and unique single-dose propositions.

• HAE market projected to reach $6.3B by 2030, ATTR market to $16.8B, with significant unmet needs and large addressable patient populations.

• Collaborations with Regeneron and other partners support pipeline expansion and commercialization.

Clinical development and data highlights

• Lonvo-z Phase 3 HAELO trial fully enrolled; topline data expected mid-2026, BLA submission in second half of 2026, and planned US launch in 1H 2027.

• Lonvo-z Phase 1/2 data show rapid, deep, and durable kallikrein reduction, with most patients attack-free and off long-term prophylaxis for up to 3 years.

• Nex-z Phase 3 trials for ATTR currently on clinical hold; Phase 1 data show rapid, deep, and durable TTR reduction, improved disease measures, and lower all-cause mortality compared to matched cohorts.