UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of DNA to be added, removed, or edited.

NTLA-2001 is designed to eliminate the TTR gene from the DNA of liver cells, which would reduce TTR production significantly and prevent the symptoms of ATTR from occurring.