Post by AIRNA

We’re excited to share new preclinical data at #ASGCT2026 highlighting the growing breadth of our RNA-editing platform, from our lead #AATD program, AIR-001, to cardiovascular research designed to introduce protective variants.   In an oral presentation, we’re featuring new in vivo data supporting AIR-001, our potential best-in-class therapeutic candidate for alpha-1 antitrypsin deficiency (AATD), which is currently being evaluated in the RepAIR1 clinical trial. Preclinical studies showed up to 59% precise RNA editing in vivo, resulting in more than 40 µM functional M-AAT and 70 µM total AAT.   We’re also presenting new data demonstrating the potential of RNA editing to introduce protective variants in two proteins linked to reduced LDL cholesterol, including findings showing up to a 92% reduction in LDL cholesterol in a humanized mouse model. Learn more in today’s press release: https://lnkd.in/euHGE6qf #RNAEditing #CardiovascularDisease #RareDisease