AIRNA has begun dosing participants in a Phase 1 clinical trial of AIR-001. Concurrent with this milestone, the company revealed that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AIR-001. Delivered via subcutaneous injection, AIR-001 is designed to correct the disease-causing PiZ mutation at the RNA level without permanently altering DNA, with the ultimate goal of restoring functional AAT protein production to treat both the lung and liver manifestations of the disease. The open-label, integrated study expects to enroll approximately 54 adult patients across an estimated 20 sites in 11 countries to evaluate the drug’s safety, tolerability, pharmacokinetics, and pharmacodynamics.
AIRNA is a clinical-stage company developing RNA-editing medicines designed to repair disease-causing genetic variants and introduce beneficial variants that promote human health. The company’s proprietary platform is based on research by RNA-editing pioneers and co-founders Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University). AIRNA is advancing a pipeline of RNA-editing therapeutics led by a product candidate for alpha-1 antitrypsin deficiency (AATD). AIRNA is headquartered in Cambridge, MA, with research operations in Tübingen, Germany.
AATD is a genetic disease that leads to progressive lung disease and liver injury. AIR-001 is designed to correct the most common disease-causing mutation in AATD (PiZ) at the RNA level. By precisely editing a specific RNA location and restoring normal AAT protein levels, AIR-001 has the potential to address both the lung and liver manifestations of AATD.
Read the full press release here.
