Intellia Therapeutics is a clinical-stage biopharmaceutical company developing in vivo CRISPR-based gene editing therapies. Rather than managing diseases with lifelong chronic therapy, Intellia aims to permanently edit specific genes in the liver with a single outpatient IV infusion, treating diseases at their root cause. Intellia has two lead candidates. Lonvo-z targets hereditary angioedema (HAE), a rare disease causing severe swelling attacks, by permanently inactivating the KLKB1 gene in the liver to eliminate the protein that triggers attacks. Lonvo-z is in Phase 3, with topline data expected by mid-2026 and a U.S. launch targeted for the first half of 2027, which would be the world's first approved in vivo CRISPR therapy. Nex-z targets ATTR amyloidosis, a fatal disease caused by misfolded TTR protein destroying the heart and nerves, by permanently knocking out the TTR gene. Nex-z is in two Phase 3 trials, though the larger ATTR cardiomyopathy trial remains on FDA clinical hold following a patient death and Grade 4 liver enzyme elevation event. Intellia is pre-revenue and funds operations through a collaboration with Regeneron, which covers ~25% of nex-z development costs in exchange for a 25% profit share. Intellia had $605M in cash as of year-end 2025, expected to fund operations into the second half of 2027. Intellia also licenses its ex vivo CRISPR platform to partners in CAR-T and autoimmune therapies, generating potential milestone and royalty streams.