High Efficiency RNA Editing Gene Therapy for the Treatment of Neurological Disorders

Time: 4:40 pm
day: Day One


  • Redirecting the fully human, endogenous ADAR enzyme enables site-specific RNA editing to correct point mutations, control RNA splicing or modulate protein expression and function. Importantly, this is done without risk of DNA damage or foreign protein immunogenicity
  • The RNAfixTM technology platform combines high throughput screening with generative machine learning models to produce highly efficient and specific ADAR-recruiting gRNAs for any clinically-relevant target, including those in sequence contexts that are naturally disfavored by the ADAR enzyme
  • Neurological disorders are particularly amenable to an RNA editing therapeutic approach due to high expression of both ADAR1 and ADAR2 enzymes
  • Application of the RNAfix technology in Parkinson’s disease will be highlighted