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In Vivo Therapies

  • A Novel Oligonucleotide-Based RNA Base Editing Therapeutic Approach for the Treatment of Hurler Syndrome

    The program uses LEAPER (Leveraging endogenous ADAR for programmable editing of RNA), a novel RNA base editing technology. Oligonucleotides of various design, called arRNA, were developed to produce precise and sequence specific A-to-I conversion in the mRNA sequence for codon 402, leading to generation of the wild type IDUA mRNA and protein.
  • In May 2020, EdiGene presented data on RNA base editing program for patients with the most severe form of mucopolysaccharidosis type I (MPS I), known as Hurler syndrome, at the 23rd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting 
  • In May 2023, EdiGene presented promising preclinical POC data for LEAPER 2.0-based in vivo RNA Editing therapies in NHP model at the 26th annual meeting of ASGCT via oral presentation