Cambridge, MA, USA and Tübingen, Germany, September 19, 2023 – AIRNA, a biotech company pioneering RNA editing therapeutics to restore the health of patients with rare and common diseases, today emerged from stealth with a $30 million initial financing led by ARCH Venture Partners. The financing enables AIRNA’s experienced team to advance a pipeline of RNA editing therapeutics driven by its powerful and flexible RNA editing platform, RESTORE+™.

“RNA editing is poised to lead the next generation of RNA therapeutics by targeting diseases not accessible through other approaches with a medicine that can be delivered to large patient populations,” said Kris Elverum, President and CEO of AIRNA. “We are privileged to work with the pioneers of the RNA editing field, as founders of AIRNA, to advance our RESTORE+™ technology platform and develop best-in-class therapeutics.”

AIRNA’s co-founders, Thorsten Stafforst, Jin Billy Li, Paul Vogel, and Tobias Merkle, were the first to publish peer-reviewed papers showing ADAR-mediated RNA editing and the use of oligonucleotides to recruit endogenous ADAR for targeted RNA editing. AIRNA’s RESTORE+™ platform is a systematic advancement of this groundbreaking research that is generating a pipeline of best-in-class products by optimizing each therapeutic’s sequence, chemistry, and delivery for precise, efficient, and safe RNA editing.

“Precisely editing RNA started as an idea in my lab over a decade ago,” said Thorsten Stafforst, co-founder of AIRNA and professor at the University of Tübingen. “After making several technological improvements, I’m excited to see AIRNA advancing RNA editing therapeutics into precise medicines that could really impact patient’s lives.”

RNA editing is a disruptive therapeutic modality with unique potential to unlock the full promise of genetically defined medicines for rare and common diseases. ADAR-directed RNA editing delivers a safe oligonucleotide that is programmed to recruit an endogenous cellular enzyme, ADAR, to introduce a precise modification to a patient’s RNA, resulting in changes to the encoded therapeutic proteins.

“AIRNA has a uniquely powerful technology to maximize the therapeutic properties of RNA editing medicines and achieve the vision of restoring a patient’s health,” said Rodger Novak, AIRNA’s Board Chair. “We now have the capability to precisely rewrite RNA’s genetic instructions, which creates the potential to address a wide range of new targets and diseases.”

As a first step, AIRNA is advancing development of a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD). AIRNA is also actively pursuing a pipeline of proprietary programs with RESTORE+ to address multiple prevalent diseases with high unmet need.

“The way AIRNA’s novel and specific approach achieves a broad range of therapeutic effects demonstrates the enduring power of deep scientific insights,” said Keith Crandell, Co-Founder and Managing Director at ARCH Venture Partners. Sean Kendall, Partner, Europe, at ARCH Venture Partners, added: “The rigor and flexibility of AIRNA’s platform shows what can be achieved when top teams collaborate across continents.”