STAT+: 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 and as long as one year, the company said.
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