GoKawiilIntellia's in-body CRISPR therapy shows lasting effect after four years
Intellia Therapeutics has released four-year follow-up data showing its gene-editing therapy lonvoguran ziclumeran continues to suppress a protein that triggers hereditary angioedema attacks. The treatment uses CRISPR-Cas9 delivered directly into the body to disable the gene producing prekallikrein, rather than editing cells outside the body as with existing therapies. Intellia has filed for FDA approval, with a decision expected by 10 March.
GoKawiil's interpretation of the reporting above, not reported fact.
If cleared, the therapy would be the first in-vivo CRISPR medicine approved anywhere, a step beyond the only currently approved CRISPR treatment, Casgevy, which requires removing and re-infusing cells. Specialists cited in the reporting suggest durable results could reduce patients' reliance on ongoing medication regimens for a lifelong condition, though long-term safety and real-world outcomes will depend on regulatory review and post-approval monitoring.
- Lonvoguran ziclumeran shows sustained suppression of swelling-related protein four years post-treatment
- It would be the first FDA-approved CRISPR therapy delivered inside the body, unlike Casgevy
- FDA decision on Intellia's application is expected by 10 March
Source: nature.com — Ledford, 2026-11-12
Published there as: “Novel CRISPR therapy is poised to gain first FDA approval to alter genes inside the body”
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