Addressing Paediatric Genetic Diseases Using a Promoterless, Nuclease-Free Gene Insertion Technology to Provide Life-Long Protein Expression

Time: 1:30 pm
day: Post-Conference Discussion Day - Track A


  • Safe and precise genome integration into the albumin gene site provides strong corrective transgene expression from the albumin promoter
  • Many life-altering and life-shortening metabolic diseases result in liver damage. GeneRide-edited hepatocytes are healthier than mutationbearing hepatocytes resulting in selective pressure to repopulate the growing and/or damaged liver with the healthy corrected cells
  • We can monitor selective advantage and expansion of corrected hepatocytes using a circulating technology-related biomarker, and we observe increasing numbers of hepatocytes expressing the corrective gene overtime, leading to reduced disease burden