RNA Editing Biotech AIRNA Secures $155M Series B to Advance Novel AATD Treatment

AIRNA, a transatlantic biotechnology company specializing in RNA editing, has successfully closed a $155 million Series B financing round. The funds will primarily support the advancement of the company's lead candidate, AIR-001, into clinical trials for the treatment of alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the lungs and liver.

AIRNA's Innovative Approach to AATD Treatment

AIR-001 employs a cutting-edge RNA editing technique to address AATD by targeting and repairing the most common pathologic mutation in the SERPINA1 gene. This approach aims to restore the expression of functional alpha-1 antitrypsin (AAT) protein, potentially offering a functional cure for AATD patients.

The technology leverages an enzyme called adenosine deaminase acting on RNA (ADAR), which is naturally present in human cells. AIRNA's platform recruits ADAR to make targeted adenosine-to-inosine edits in RNA, effectively correcting the genetic defect responsible for AATD.

AIRNA CEO Kris Elverum emphasized the appeal of AIR-001, stating, "Patients and physicians want to be able to have options that can give them a functional cure while maintaining flexibility and control over their healthcare decisions."

Clinical Trial Plans and Pipeline Development

With the new funding, AIRNA plans to initiate a Phase I/II clinical trial for AIR-001 in the second half of 2025. The company has completed in vivo proof-of-concept studies for the candidate and is preparing to submit regulatory applications for clinical trial approval.

In addition to advancing AIR-001, the Series B proceeds will support the development of AIRNA's pipeline of other RNA-editing candidates. The company is exploring applications in cardiometabolic diseases and other genetic conditions, focusing on both disease-causing variations and potentially beneficial genetic variants.

Investment Landscape and Industry Context

The Series B round was co-led by Venrock Healthcare Capital Partners and Forbion Growth, with participation from institutional investors such as ARCH Venture Partners, RTW Investments, and Nextech Invest. This successful fundraising effort comes amid a challenging macro-environment for the biotech industry, highlighting the continued investor interest in innovative therapeutic approaches.

AIRNA's raise follows recent funding successes by other biotechs, including Hillstar Bio's $67 million Series A for autoimmune therapies and Temper Bio's $70 million Series B for substance-use disorder treatments.

The RNA editing field has attracted significant attention and investment, with companies like Wave Life Sciences, Shape Therapeutics, and Korro Bio also developing similar technologies. However, the long-term clinical success and market positioning of these approaches remain to be determined.