Intellia Therapeutics shows improved, sustained effect from in vivo CRISPR treatment

Intellia Therapeutics said Monday that its CRISPR-based therapy for a genetic nerve disorder reduced the levels of a disease-causing protein by 93% in a new group of patients treated at the highest dose used to date — surpassing initial, lower-dose results reported last June.

The one-time Intellia therapy, called NTLA-2001, is also showing a sustained effect, with protein reductions remaining stable in previously treated patients followed for nine months, the company said.

The updated study results reported Monday remain preliminary but provide further evidence that NTLA-2001 can be delivered systemically via a one-time intravenous infusion and safely edit the damaged DNA of liver cells inside the body.

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