ADVY NEWS "Breakthrough CRISPR Milestone: For the first time Intellia Therapeutics successfully reports In Vivo Genome editing"
In a major milestone for the still-young field of genome editing, Intellia Therapeutics said 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.