CRISPR/Cas9 Gene Editing in Hemophilia B

Time: 9:30 am
day: Day One


  • A landmark phase 1 clinical trial demonstrated successful gene editing for transthyretin amyloidosis, establishing proof of concept for CRISPR-Cas9 treatment in humans (Intellia Therapeutics and Regeneron Pharmaceuticals)
  • Intellia Therapeutics and Regeneron Pharmaceuticals are developing a targeted insertion platform for the expression of Factor IX from the Albumin locus in liver for the treatment of Hemophilia B, with pre clinical data showing robust and durable FIX expression in mouse and non-human primates
  • The Factor IX gene insertion program for the treatment of Hemophilia B is currently advancing toward IND-enabling studies