AIRNA

AIRNA is a biotechnology firm specializing in the development of RNA editing therapeutics aimed at treating both rare and common diseases. The company was founded in 2021 by a team of scientists including Thorsten Stafforst, Jin Billy Li, Paul Vogel, and Tobias Merkle, and it emerged from stealth mode in September 2023 with an initial $30 million financing led by ARCH Venture Partners. The founders are pioneers in the field; Stafforst, a professor at the University of Tübingen, and Li, an associate professor at Stanford University, were the first to publish research demonstrating ADAR-mediated RNA editing. Their extensive research, dating back over a decade, forms the scientific backbone of the company.

The company's business is centered on its proprietary RESTORE+ platform, which engineers oligonucleotides to recruit an endogenous enzyme called ADAR (adenosine deaminases acting on RNA). This process introduces a precise modification to a patient's RNA, correcting genetic mutations at the transcript level without altering the underlying DNA. This approach offers a key advantage by being reversible and allowing for convenient re-dosing, distinguishing it from permanent gene-editing techniques like CRISPR. AIRNA's business model involves leveraging this platform to build a pipeline of proprietary drug candidates. The company's funding is venture capital-based, having raised a total of $245 million through multiple rounds, including a $155 million Series B in April 2025, to advance its programs into clinical trials.

AIRNA's primary service is the discovery and development of these RNA editing medicines. Its lead product candidate, AIR-001, targets alpha-1 antitrypsin deficiency (AATD), a genetic disorder that can cause severe lung and liver disease. The therapy is designed to repair the most common mutation in the SERPINA1 gene to restore the production of functional protein. A key feature of their delivery system is the use of GalNAc conjugation, which specifically targets liver cells and avoids the use of lipid nanoparticles (LNPs), potentially offering a better safety profile. The company plans to file for a Phase 1/2 clinical trial for AIR-001 in the second half of 2025. Beyond AATD, AIRNA is actively developing a pipeline to address other conditions, including cardiometabolic and neurodegenerative diseases.

Keywords: RNA editing, therapeutics, ADAR, RESTORE+ platform, gene therapy, genetic medicine, oligonucleotide therapeutics, alpha-1 antitrypsin deficiency, AATD, genetically defined diseases, Thorsten Stafforst, Jin Billy Li, Kris Elverum, ARCH Venture Partners, Forbion, Venrock, rare diseases, cardiometabolic disease, biopharmaceutical, site-directed RNA editing, preclinical, clinical trials, SERPINA1