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

CAMBRIDGE, Mass. & TÜBINGEN, Germany--(BUSINESS WIRE)-- 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+^TM.

“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.”

AIRNA’s Leadership and Founders

Kris Elverum, President and Chief Executive Officer, AIRNA, was previously CEO of Diagon Therapeutics, and held various senior management positions at Rubius Therapeutics, Turnstone Biologics and SQZ Biotech.

Sriram Sathyanarayanan, PhD, Chief Scientific Officer, AIRNA, was previously CSO at Codiak BioSciences, and held leadership positions at Jounce and Merck.

Rodger Novak, MD, Board Chair, AIRNA, was co-founder and former Chairman and CEO of CRISPR Therapeutics, and serves on multiple biotech Boards.

Paul Vogel, PhD, Founder and Vice President, AIRNA, has over 10 years of experience in site-directed RNA editing, studying in the labs of our scientific founders.

Tobias Merkle, PhD, Founder, Senior Director, Platform at AIRNA, has 9 years RNA editing research experience and previously was a senior research scientist at Roche.

Thorsten Stafforst, PhD, Founder, Professor at University of Tübingen has been pioneering ADAR-mediated RNA base editing since 2011 and was the first to publish on the field in peer review. Thorsten has co-authored more than 40 peer reviewed publications.

Jin Billy Li, PhD, Founder, Associate Professor at Stanford University, has been studying ADAR-mediated RNA editing since 2006, and his lab has made significant contributions to ADAR-related biology and engineering. Billy has co-authored 83 peer reviewed publications.

About ARCH Venture Partners

ARCH Venture Partners creates and invests in ground breaking life science and technology companies. The firm is a recognized leader in commercializing technologies developed at academic institutions, corporate research groups and national laboratories. ARCH invests primarily in companies it co-founds with leading scientists and entrepreneurs, bringing innovations in life sciences and physical sciences to market. For more information, visit www.archventure.com.

About AIRNA

AIRNA is pioneering the discovery and development of RNA editing therapeutics to deliver on the promise of genetically defined medicines for patients with rare and common diseases. 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 conveniently re-dosed and manufactured. AIRNA’s founders, Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University), were the first to elucidate a therapeutic approach for precise editing of RNA. AIRNA has advanced this groundbreaking research to establish the RESTORE+^TM RNA editing platform to restore patients’ health.

AIRNA is advancing its first therapeutic program, a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD), as well as a pipeline of therapeutic candidates to address multiple diseases with high unmet need. Initial financing of the company was led by ARCH Venture Partners, with participation from ND Capital, Fast Track Initiative (FTI), Novalis, and Codon Capital. AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen, Germany. Further information can be found at www.airna.com.

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