- In vivo data support potential best-in-class profile for AIR-001, currently in Phase 1 clinical trial for alpha-1 antitrypsin deficiency (AATD)
- Data from cardiometabolic portfolio demonstrate promise of AIRNA’s approach to introduce beneficial variants
CAMBRIDGE, Mass.--(BUSINESS WIRE)--AIRNA, a biotech company pioneering RNA-editing therapeutics to transform the lives of patients with rare and common conditions, will present new preclinical data from the company’s pipeline of RNA-editing therapeutics, including lead candidate AIR-001 and the company’s cardiometabolic portfolio, at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
In an oral presentation, AIRNA will present the first in vivo data on AIR-001, AIRNA’s lead RNA-editing therapeutic candidate for alpha-1 antitrypsin deficiency (AATD). AIR-001 is a subcutaneously delivered GalNAc oligonucleotide designed to correct the most common disease-causing mutation of AATD (PiZ) at the RNA level by recruiting endogenous ADAR enzymes to precisely edit SERPINA1 mRNA. AIR-001 is currently being evaluated in the Phase 1 RepAIR1 clinical trial (NCT07431112).
Additionally, in a poster at ASGCT, the company will share preclinical proof-of-concept studies utilizing RNA editing to introduce naturally occurring protective variants in the transcripts of two genes implicated in cardiovascular disease: low-density lipoprotein receptor (LDLR) and apolipoprotein B (APOB). These findings highlight the breadth of AIRNA’s RNA-editing platform, which has the potential to precisely introduce beneficial changes at the RNA level.
Details of the presentations are as follows:
- Oral Presentation Title: “AIR-001: A highly potent and precise GalNAc-conjugated RNA base editing treatment for alpha-1 antitrypsin deficiency”
- Abstract Number: 77
- Session: Translational Gene Therapy and Editing in Storage Disorders
- Date/Time: Tuesday, May 12, 11:15 – 11:30am ET
- Location: MCEC Room 258ABC
- Poster Presentation Title: “RNA Editing: A Modality for Precise Introduction of Gain-of-Function Variants Identified by Human Genetics”
- Abstract Number: 1426
- Session: Poster Reception
- Date/Time: Tuesday, May 12, 5 pm - 6:30 pm
- Location: MCEC Exhibit and Poster Hall B2-C, Exhibit level
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/.
Contacts
Media:
Michael Galfetti
Ten Bridge Communications
tbcAIRNA@tenbridgecommunications.com
