Sangamo to Begin First Human Trial of in vivo Gene Editing

Sangamo Therapeutics has announced that they are ready to start the first-ever trial of gene editing in the human body with a study of gene therapy for hemophilia B.  All hemophilia B gene therapy studies so far have inserted new factor IX genes into the nucleus of cells in the body, but those new genes have intentionally not been integrated into the genome of the cells.  Sangamo will use molecules called zinc finger nucleases to actually insert the new factor IX gene into a location on the cell’s chromosomes.  Sangamo plans to insert the new factor IX gene next to the albumin gene in liver cells where it would be under the control of the albumin gene’s regulatory elements.  Since the body produces large amounts of albumin, the hope is that it will also produce factor IX the same way.  Sangamo’s Phase I study is currently recruiting patients.  More information can be found on www.clinicaltrials.gov under study identifier NCT02695160.