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In a major milestone for the still-young field of genome editing, Intellia Therapeutics said Saturday that the first six patients to receive a CRISPR-based treatment for a genetic nerve disorder have safely had the DNA inside their liver cells edited.

Preliminary results from the study — the first to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body  — found that the treatment reduced levels of a disease-causing protein by an average of 87% in the higher dose cohort with only mild side effects. The encouraging interim Phase 1 results, presented at a conference on Saturday, were published simultaneously in the New England Journal of Medicine.


“This reflects what we’ve done preclinically where we see these very nice dose-dependent effects where at the higher doses we’re getting to virtually every single hepatocyte,” said Laura Sepp-Lorenzino, Intellia’s chief scientific officer. The Intellia therapy, called NTLA-2001, is being co-developed by Regeneron Pharmaceuticals.

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