AIRNA develops RNA‑editing medicines using a proprietary platform founded on academic research to precisely edit target RNA for both rare and common diseases. Its lead program, AIR‑001, is designed to repair the most common harmful SERPINA1 (PiZ) mutation in alpha‑1 antitrypsin deficiency (AATD) to restore functional M‑AAT production, with subcutaneous dosing and a focus on potency, durability, and safety. The company plans to file a clinical trial application for AIR‑001 in 2H 2025 and advance the candidate into a Phase 1/2 study. Proceeds from the recent financing will also fund development of a pipeline targeting cardiometabolic and other diseases by introducing beneficial genetic variants or repairing harmful ones. AIRNA cites research origins from University of Tübingen and Stanford co‑founders and maintains headquarters in Cambridge, MA, with research operations in Tübingen, Germany. AIRNA is developing RNA editing therapeutics via its RESTORE+ platform, which optimizes oligonucleotide sequence, chemistry, and delivery for precise RNA editing. Its lead product candidate is positioned as a potential best-in-class treatment for alpha-1 antitrypsin deficiency (AATD). The company is advancing a pipeline of candidates to address multiple diseases with high unmet need. AIRNA plans to bring its first drug candidate to the clinic next year and will use Series A proceeds to advance that program and further develop the platform. The company launched with backing from ARCH Venture Partners based on research by academic co-founders Thorsten Stafforst and Jin Billy Li. AIRNA is headquartered in Cambridge, MA, with research operations in Tübingen, Germany, and has raised a total of $90 million in Series A funding. AIRNA emerged from stealth to develop a new class of RNA editing therapeutics built on its RESTORE+ platform, which programs oligonucleotides to recruit endogenous ADAR for precise RNA modifications. The company’s scientific founders were among the first to publish ADAR-mediated RNA editing and the use of oligonucleotides to recruit endogenous ADAR for targeted editing. AIRNA is advancing a pipeline of candidates by optimizing sequence, chemistry, and delivery to achieve precise, efficient, and safe RNA editing. Its initial development program is a candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD), and the company is pursuing additional proprietary programs for prevalent diseases with high unmet need. AIRNA says its approach enables medicines that can be conveniently re-dosed and manufactured for large patient populations. The company is headquartered in Cambridge, MA, with research operations in Tübingen, Germany.

Total raised

$245M

Funding rounds

3

Latest round

Series B

Latest activity

Apr 2025