Intellia's lonvo-z hit primary in the HAELO Phase 3 on April 27, 87 percent reduction in hereditary angioedema attacks over six months from a single intravenous infusion. No apheresis, no conditioning chemo, no hospital admission. The first global Phase 3 readout for in vivo CRISPR. BLA queued for the second half of this year, US launch slated for first-half 2027.

The analyst desks priced in vivo gene editing on one assumption. Casgevy worked because the cells came out, got edited under laboratory control, and went back in after myeloablative chemo cleared the marrow. Anything looser was a delivery problem the field had failed to crack in ten years. Lipid nanoparticles to hepatocytes were a partial answer, durability past Phase 1 was unproven, and a one-shot edit on an outpatient drip got treated as a research story years from any commercial pathway.

Three-year Phase 1 durability and the pivotal both landed inside one fiscal year. Intellia's second in vivo Phase 3, nex-z for transthyretin amyloidosis, is reading out on the same chemistry. The HAE prophylaxis franchises Takeda, BioCryst, and CSL traded as decade-long annuities get marked as runoff against a single-dose cure.

LNP-to-liver delivery is the platform. The queue behind these two indications runs the whole monogenic liver list, alpha-1 antitrypsin, familial hypercholesterolemia, hemophilia B, every condition gated on reaching a hepatocyte without sending the patient through a transplant ward. The platform just cleared its first pivotal.