RARE Daily

AIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases

September 19, 2023

Rare Daily Staff

AIRNA, a company pioneering RNA editing therapeutics to restore the health of patients with rare and common diseases, has emerged from stealth with a $30 million initial financing led by ARCH Venture Partners.

The financing enables AIRNA’s 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.

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 (adenosine deaminases acting on RNA) 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.

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. AIRNA is also actively pursuing a pipeline of proprietary programs with RESTORE+ to address multiple prevalent diseases with high unmet need.

Photo: Kris Elverum, president and CEO of AIRNA

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