Intellia Therapeutics

Type: Company (clinical-stage in vivo gene editing) Ticker: NTLA (Nasdaq) Co-founded by: Jennifer Doudna (CRISPR Nobel laureate) among others

Intellia is the company that took in vivo CRISPR — editing a gene directly inside the patient's body via a single LNP infusion — from concept to the first Phase 3 success for the modality. Its lead asset, lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002), treats hereditary angioedema (HAE) by permanently inactivating the liver KLKB1 (kallikrein B1) gene, lowering the kallikrein/bradykinin that drives HAE swelling attacks.

Key Contributions

• HAELO Phase 3 success (Apr 2026) — Lonvo-z cut HAE attacks 87% vs placebo (p<0.0001); mean monthly attacks 0.26 vs 2.10; 62% attack-free vs 11%; single 50 mg dose; 80 patients; no serious adverse events in the treatment arm. A global first for in vivo gene editing at Phase 3. (Intellia HAELO Phase 3)
• Rolling BLA + 1H 2027 launch target — Rolling BLA submission initiated with the FDA; completion expected 2H 2026, US launch anticipated 1H 2027. (Intellia HAELO Phase 3)
• In vivo LNP-to-liver knockout platform — Validates the systemic-infusion, gene-knockout playbook (vs ex vivo Casgevy), the template Verve and Beam also pursue. (Intellia HAELO Phase 3)

Mentioned In

• CRISPR Clinical Translation — lonvo-z is the field's first in vivo Phase 3 win.

Related Entities

• Cleveland Clinic — ran the parallel in vivo CRISPR cholesterol (ANGPTL3) trial; same in vivo LNP-to-liver paradigm.
• Beam Therapeutics — base-editing counterpart pursuing in vivo correction rather than knockout.