STAT+: Intellia says CRISPR treatment safely corrects DNA of six patients with rare disease

Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells.

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body — found that the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively. In the low-dose group, the one-time infusion also reduced by 91% the painful swelling “attacks” commonly experienced by patients with a rare condition called hereditary angioedema, or HAE. Participants in the high-dose group have not yet completed the 16-week observation period.

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