LEAPER 2.0 agents

LEAPER 2.0 (Leverage Endogenous ADAR for Programmable Editing on RNA) is a second-generation RNA editing technology platform developed by EdiGene. The platform utilizes adeno-associated virus (AAV) vectors to deliver circular ADAR-recruiting RNAs (arRNAs), which are designed to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to specific RNA transcripts. This recruitment enables precise adenosine-to-inosine (A-to-I) editing to correct pathogenic mutations, such as G-to-A transitions or premature stop codons. Compared to the first-generation LEAPER technology, LEAPER 2.0 offers significantly enhanced editing efficiency, improved specificity, and greater stability due to the circular structure of the arRNA, which protects it from exonuclease-mediated degradation. The technology has demonstrated therapeutic potential in humanized mouse models of Hurler syndrome (MPS IH) by successfully restoring IDUA enzyme activity.

Other names

LEAPER 2.0LEAPER2.0LEAPER-2.0Leverage Endogenous ADAR for Programmable Editing on RNA 2.0