Milestones
- First patient dosed2021Complete.
- Phase 2 results published in NEJMOct 2024Complete.
- Phase 3 fully enrolled2025Complete.
- Phase 3 headline: 87% fewer attacks vs placebo27 Apr 2026Complete.
- Full results in NEJM and at EAACIJun 2026Complete.
- FDA accepts filing with priority review8 Sep 2026Complete.
- US commercial launchTarget 2027Current milestone.
Most important updates
- 8 Sep 2026
- 13 Jun 2026
- 27 Apr 2026
- Feb 2024
- Sep 2022
Upcoming
- 2027US commercial launch (next)
- 2027Planned US launch of lonvo-z
- 10 Mar 2027FDA decision date (PDUFA) for lonvo-z in hereditary angioedema
Current obstacles
- Long-term safetyGood preventive drugs already exist, so regulators will weigh a permanent edit against limited long-term data.
- How to pay for a one-time curePricing a single treatment that replaces years of ongoing drugs is untested at scale.
Physics limits
- Fat-bubble delivery mostly reaches the liverIn the blood, lipid nanoparticles pick up a protein (ApoE) that liver cells absorb. That makes the liver easy to edit, but most other organs (brain, muscle, lungs) remain hard to reach with enough dose.
- Edits in the body are permanent and can't be recalledOnce liver cells are edited, the change lasts for life and is copied as cells renew. Any off-target cut or unexpected long-term effect can't be undone, so safety must be proven over years.
- Guides can bind near-matching DNA elsewhereA 20-letter guide can tolerate a few mismatches, so the editor may act at similar sites among 3 billion letters. Screening reduces but can't fully rule out rare off-target edits in every cell.
How it works
1. Infuse
Drip to the liver
A single infusion of fat nanoparticles carries the CRISPR editor's instructions and guide; liver cells naturally take them up from the blood.
2. Cut
Switch off one gene
CRISPR breaks the KLKB1 gene, so the liver stops making the protein that starts the swelling chain.
3. Effect
Fewer attacks
Less of that protein means less of the signal (bradykinin) that causes swelling attacks.
Spec sheet
| Spec | Lonvo-z (NTLA-2002) |
|---|---|
| Target gene | KLKB1 (prekallikrein)R (reported) |
| Delivery | LNP, single IV infusionR (reported) |
| Phase 3 dose | 50 mgR (reported) |
| Attack reduction vs placebo | 87% (mean monthly attacks)R (reported) |
| Attack-free over 6 months | 62% of treated patientsR (reported) |
R reported by the company
Papers & demos
- Jun 2026paperHAELO Phase 3 results for lonvo-z (NEJM, presented at EAACI 2026) (opens doi.org)First successful Phase 3 trial of an in-body gene-editing drug.
- Oct 2024paperCRISPR-based therapy for hereditary angioedema, Phase 2 (NEJM) (opens doi.org)Controlled trial showing one dose of lonvo-z cut attacks vs placebo.
Update log
Tue 8 Sep
- Major: PressRegulatory
Sat 13 Jun
- Minor: PaperResearch
Mon 27 Apr
- Major: FilingTest
Feb 2024
- Minor: PaperResearch
Sep 2022
- Minor: PressTest
About Intellia Therapeutics
Intellia Therapeutics
In-body CRISPR, lonvo-z, nex-z
Company co-founded by Jennifer Doudna that pioneered CRISPR inside the body. One drip carries the editor to the liver to switch off a disease gene for good. Lonvo-z, for hereditary angioedema, passed Phase 3; its ATTR drug resumed after a 2025 safety hold.
- Phase 3 (Apr 2026): lonvo-z cut swelling attacks 87% vs placebo; 62% had none. Filing with the FDA for a 2027 launch.
- Oct 2025: a patient had severe liver injury and later died; the FDA paused nex-z trials. They restarted in 2026 with more liver checks.
- In Jan 2025 Intellia dropped outside-the-body editing and cut staff to focus on its two liver drugs.
- Founded
- 201412 yrs
- Headquarters
- United States
- Status
- Public
- Listed
- NTLA (opens google.com)NASDAQ
- Valuation
- public
- Works in
- BiotechGene editing
- Coverage
- 2 programs · 11 updateslatest 8 Sep 2026checked 25 Sep
- Partners
- Regeneron (opens regeneron.com)
- People
- John Leonard (opens intelliatx.com)CEOJennifer Doudna (opens en.wikipedia.org)Co-founder