AIRNA Presents New Preclinical Data on AATD Therapeutic Candidate AIR-001 and Cardiometabolic RNA-Editing Programs at ASGCT

- AIR-001 demonstrated best-in-class potential, achieving up to 59% precise RNA editing in vivo and restoring 40 µM functional M-AAT in a mouse model of AATD

- Preclinical data show RNA editing can introduce protective variants for cardiovascular disease and drive reduction of LDL cholesterol levels

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AIRNA, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common conditions, is presenting new preclinical data on its therapeutic candidate AIR-001 for the treatment of alpha-1 antitrypsin deficiency (AATD) and its cardiometabolic RNA-editing research programs at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Boston from May 11-15, 2026. Together, the presentations demonstrate the potential of AIRNA’s RNA-editing platform both to repair disease-causing variants and to introduce protective variants associated with human health.

“We are excited to showcase for the first time our robust preclinical data for AIR-001 — now being evaluated in the clinic — as well as new data that illustrate the breadth of AIRNA’s RNA-editing platform,” said Sriram Sathy, Ph.D., Chief Scientific Officer of AIRNA. “From repairing disease-causing variants to introducing protective variants, we believe RNA editing offers a powerful and flexible approach to therapeutic development.”

Preclinical data on AIR-001

New data being presented at ASGCT include the first in vivo data on AIR-001, AIRNA’s RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD), which is currently being evaluated in a Phase 1 clinical trial (NCT07431112). AATD is a genetic disease caused by mutations in the SERPINA1 gene that reduce levels of functional alpha-1 antitrypsin (M-AAT) protein, leading to progressive lung disease and liver injury. AIR-001, a subcutaneously delivered GalNAc oligonucleotide, is designed to correct the most common disease-causing mutation of AATD (PiZ) at the RNA level.

In a mouse model of AATD, subcutaneous delivery of AIR-001 led to potent and precise editing of the PiZ mutation with no off-target edits observed. Liver RNA editing showed dose-dependent effects, with the 10 mg/kg dose achieving 59% editing, corresponding with >40 µM of corrected alpha-1 antitrypsin (M-AAT) in serum and 70 µM total AAT. A longer-term study in mice assessing AIR-001 delivered bi-weekly for 15 weeks found that it drove accumulating levels of M-AAT and total AAT, indicating a potential for long-term benefit in patients. AIR-001 also demonstrated improvement across lung and liver disease-relevant endpoints, including >30-fold increase in neutrophil elastase inhibition. Finally, in non-human primate studies, AIR-001 demonstrated tolerable and durable liver exposure, supporting the potential for a dosing regimen of once every 8-12 weeks in humans.

Cardiometabolic portfolio

In a poster presentation at the meeting, AIRNA is sharing preclinical proof-of-concept data demonstrating the potential of RNA editing to introduce protective variants in cardiovascular disease targets, with particularly strong findings for low-density lipoprotein receptor (LDLR).

Based on human genetics studies identifying a protective variant in the LDLR gene associated with substantially lower LDL cholesterol, AIRNA developed an RNA editing oligonucleotide designed to replicate the effect of this variant at the RNA level. In human hepatocytes, the editor achieved up to 70% editing and a 3.5-fold increase in LDLR expression. In a humanized mouse model, treatment resulted in a 92% reduction in LDL cholesterol. Combination dosing with alirocumab, an approved anti-PCSK9 antibody, also produced greater LDL cholesterol reduction at lower doses than alirocumab alone, supporting the potential for use as a monotherapy as well as alongside existing lipid-lowering therapies.

AIRNA also explored apolipoprotein B (ApoB), a validated cardiovascular disease target where prior therapeutic approaches have been limited by hepatotoxicity. Employing large-scale genetic studies, researchers identified a variant in the APOB gene associated with lower LDL cholesterol in the absence of liver-related health issues. Using RNA editing to replicate the effect of this protective variant, AIRNA achieved 30% editing in vivo, a 21% decrease in ApoB protein and a 17% reduction in LDL cholesterol, without observed hepatotoxicity.

Together, these findings illustrate how AIRNA’s platform may enable the translation of naturally occurring protective variants identified through human genetics into new therapeutic approaches.

About AIR-001

AIR-001 is AIRNA’s lead RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD), a genetic disease most commonly caused by the PiZ mutation in the SERPINA1 gene. AIR-001 is designed to repair the underlying cause of disease and restore production of functional alpha-1 antitrypsin protein, with the goal of addressing both the lung and liver manifestations of AATD. Administered via subcutaneous injection, AIR-001 is designed for convenient, repeat dosing. AIR-001 has been granted Orphan Drug Designation for the treatment of alpha-1 antitrypsin deficiency from the U.S. Food and Drug Administration and is currently being evaluated in the Phase 1 RepAIR1 clinical trial (NCT07431112).

About AIRNA

AIRNA is a clinical-stage company developing RNA-editing therapeutics 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. Learn more at https://airna.com/.