AIRNA Presents Data Supporting RNA Editing Approach for Alpha-1 Antitrypsin Deficiency (AATD) at 28th ASGCT Annual Meeting

AIRNA, a biotech company pioneering RNA editing therapeutics to transform the lives of patients with rare and common conditions, today announced preclinical proof-of-concept data supporting its best-in-class RNA editing strategy for alpha-1 antitrypsin deficiency (AATD). These data are being presented May 14 from 5:30 – 7 PM CT during a poster session (#1106) at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans.

“AIRNA’s RESTORE+ platform was designed to rapidly develop potent, convenient, and safe RNA editing therapeutics for both rare and common diseases,” said Sriram Sathy, Ph.D., Chief Scientific Officer of AIRNA. “These data demonstrate the potential to provide patients suffering from AATD a best-in-class therapeutic with potent and durable wild-type M-AAT protein production, well-tolerated safety profile, and subcutaneous dosing.”

AATD is an inherited condition leading to lung and liver disease that is caused by insufficient levels of functional alpha-1 antitrypsin (AAT) protein due to mutations in the SERPINA1 gene. AIRNA’s lead RNA editing candidate is designed to precisely and potently repair the most common and harmful underlying genetic mutation (PiZ) to restore production of the functional, wild-type version of the AAT protein, known as M-AAT.

In a poster presentation entitled “Optimized RESTORE+ oligonucleotides for an efficacious and safe RNA base editing treatment for alpha-1 antitrypsin deficiency,” AIRNA’s rAIR-100 research candidates demonstrated greater than 90% editing in primary mouse hepatocytes in vitro, and greater than 50% editing in vivo in a mouse model of AATD with subcutaneous GalNAc delivery. rAIR-100 restored wild-type M-AAT protein levels above 30 μM and improved key markers of lung and liver health, increasing neutrophil elastase inhibition by over 17-fold and reducing large liver aggregates by 9-fold. Pharmacokinetics (PK) and safety in non-human primates (NHPs) showed prolonged liver exposure compared to mice, with no observable toxicity. AIRNA’s lead RNA editing product candidate, AIR-001, was further optimized for potency and durability, and AIRNA expects to file a clinical trial application in 2H 2025.

“AIRNA’s RESTORE+ platform effectively engages the endogenous isoforms of the RNA editing enzyme, ADAR, and significantly improves in vivo editing,” said Thorsten Stafforst, Ph.D., co-founder of AIRNA and professor at the University of Tübingen. “Just six years after we first published the use of engineered antisense oligonucleotides to precisely edit RNA, AIRNA’s RESTORE+ platform is demonstrating unique potential to repair disease-driving mutations, such as those causing AATD, and to introduce beneficial forms of RNA edits to improve health across broad populations.”

About AIRNA

AIRNA is harnessing advances in genetics to develop transformative RNA-editing medicines that improve human health across both rare and broad populations. RNA editing offers the precision of genetic medicine approaches while maintaining treatment convenience, flexibility, and reversibility. Our proprietary platform is based on groundbreaking research by pioneers of the field and company co-founders Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University) and enables optimal potency, safety, and delivery.

AIRNA is advancing a robust pipeline of therapeutic candidates that are designed to provide functional cures for severe or chronic diseases by repairing harmful genetic variants or introducing beneficial variants that promote optimal health. AIRNA’s lead program has the potential to be a best-in-class therapeutic for alpha-1 antitrypsin deficiency (AATD). AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen, Germany. Learn more at https://airna.com/.

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