Airna announced that the first patient has been dosed in the global Phase 1 clinical trial of AIR-001, its RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD).
The open-label RepAIR1 study (NCT07431112) is an integrated single ascending and multiple dose trial in adults with the PiZZ genotype designed to evaluate safety, pharmacokinetics and pharmacodynamics, including changes in total AAT, functional AAT, Z-AAT and M-AAT, and is planned to enroll approximately 54 patients.
The study has received authorization from regulatory authorities in multiple countries, is currently enrolling in Australia and the United Kingdom, and is expected to expand to an estimated 20 sites across 11 countries.
The U.S. Food and Drug Administration has granted orphan drug designation to AIR-001. AIR-001 is a subcutaneously delivered GalNAc oligonucleotide designed to recruit endogenous ADAR enzymes to edit SERPINA1 mRNA and correct the PiZ mutation, restoring functional AAT; the company claims the approach is reversible and repeatable and could address both lung and liver manifestations of AATD.
The company describes AIR-001 as a potential best-in-class candidate and cites preclinical data indicating it may increase functional AAT levels.
