AIRNA Raises $155 Million to Advance AATD Therapy

Rare Daily Staff

RNA editing therapeutics developer AIRNA said it raised $155 million through an oversubscribed series B financing to advance its experimental therapy AIR-001 for alpha-1 antitrypsin deficiency and develop a pipeline of novel RNA therapies.

Venrock Healthcare Capital Partners and Forbion Growth co-lead the round with participation from RTW Investments, Nextech Invest, and other new investors. Existing investors ARCH Venture Partners, Forbion Ventures, ND Capital, and others also participated.

Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the SERPINA1 gene that lead to insufficient levels of functional alpha-1 antitrypsin (M-AAT) protein, which results in lung and liver disease. AIR-001 precisely repairs the most common, harmful SERPINA1 mutation (PiZ) to address the underlying cause of both lung and liver disease and restore functional M-AAT production. AIR-001 was designed for potent and durable M-AAT production, convenient, subcutaneous dosing, and well-tolerated safety to give patients a preferred therapeutic option.

AIRNA’s proprietary platform harnesses natural mechanisms to potently edit a target RNA with a safe and flexible medicine. Precise RNA editing has unique potential to safely introduce beneficial genetic variants that promote optimal health, in addition to repairing disease-driving mutations, such as those causing AATD.

“AIRNA is developing a new class of genetic medicines that could provide functional cures for a wide range of diseases,” said Kris Elverum, president and CEO of AIRNA. “This partnership of new and existing investors allows us to rapidly deliver AIR-001 to patients with AATD, as well as progress a pipeline of medicines to realize the full potential of RNA editing.”