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Intellia files first in-vivo CRISPR therapy with the FDA

Intellia files first in-vivo CRISPR therapy with the FDA

Phase 3 HAELO cut hereditary-angioedema attacks 87%; rolling BLA for lonvo-z opens the path to the first marketed in-body gene edit

AI·Biosecurity· pending-decision How Life Changes·The Long Game ·12 takes · ·rbtfl upd 2026년 6월 24일

Summary

Intellia Therapeutics began a rolling FDA Biologics License Application on 27 April 2026 for lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002), a one-time CRISPR edit of the KLKB1 gene to permanently lower kallikrein and bradykinin in hereditary angioedema. It is the first in-vivo CRISPR therapy to reach a US filing. In the 80-patient Phase 3 HAELO trial, lonvo-z cut attack rates 87% versus placebo (weeks 5–28); 62% of treated patients were completely attack-free and off prophylaxis, with no serious or grade ≥3 treatment-emergent events. The NEJM manuscript was published alongside June's EAACI Istanbul presentation. Intellia plans to complete the BLA in H2 2026 and launch in H1 2027 if approved.

By the numbers

  • 87%, reduction in HAE attack rate vs placebo (weeks 5–28).
  • 62%, patients completely attack-free and off prophylactic therapy.
  • 80, patients in the placebo-controlled Phase 3 HAELO trial.
  • 1, number of doses (one-time treatment).
  • H1 2027, planned US commercial launch if approved.

Why it matters

A marketed in-body gene edit would shift CRISPR from one-off ex-vivo and bespoke cases to a repeatable, single-dose product, and set the regulatory template for permanent edits of common-ish disease genes. The KLKB1/liver target is the proof case; approval would validate the lipid-nanoparticle in-vivo delivery model Intellia and rivals are built on.

What to watch

  • BLA completion (H2 2026) and any FDA advisory-committee scheduling.
  • Long-term durability/safety data beyond the ~7.5-month median follow-up.
  • Whether the precedent accelerates in-vivo filings from Beam, Verve and others.