CAMBRIDGE, Mass. — AIRNA said it has dosed the first patient in a global Phase 1 clinical trial of AIR-001, an RNA-editing therapy being developed for the treatment of alpha-1 antitrypsin deficiency (AATD), marking the company’s transition into clinical-stage development.
The company also said the U.S. Food and Drug Administration has granted Orphan Drug Designation to AIR-001 for AATD, a rare genetic condition that affects the lungs and liver.
AIR-001 is designed to correct the most common disease-causing mutation in AATD at the RNA level. The therapy uses a GalNAc oligonucleotide delivered via subcutaneous injection to recruit ADAR enzymes that edit the SERPINA1 messenger RNA, with the goal of restoring production of functional alpha-1 antitrypsin protein. Unlike gene editing, the approach does not permanently alter DNA and is intended to be reversible and repeatable.
“RNA editing has the potential to deliver functional cures to people living with inherited diseases like AATD,” said Jacob S. Elkins, M.D., chief medical officer of AIRNA. “Promising preclinical data indicate AIR-001 has the potential to increase functional AAT levels to address both the lung and liver manifestations of AATD, with a reversible and repeatable dosing approach. We are excited to carry this momentum forward in our RepAIR1 global clinical study and begin translating these findings into meaningful outcomes for patients.”
AATD is caused by mutations in the SERPINA1 gene that reduce levels of functional alpha-1 antitrypsin protein, leading to progressive lung disease and liver damage.
The Phase 1 study, known as RepAIR1, is an open-label trial evaluating safety, pharmacokinetics, and pharmacodynamics in adults with the PiZZ genotype, the most severe form of the disease. The trial is expected to enroll about 54 patients and has received regulatory clearance in multiple countries.
AIRNA said the study is currently enrolling participants in Australia and the United Kingdom and is expected to expand to roughly 20 sites across 11 countries.
