Cambridge, MA, and Tübingen, Germany – AIRNA, a biotechnology company specializing in RNA editing therapeutics, has raised $155 million in an oversubscribed Series B funding round.
The financing, led by Venrock Healthcare Capital Partners and co-led by Forbion Growth, will support the clinical development of AIRNA’s lead candidate for Alpha-1 Antitrypsin Deficiency (AATD) and the expansion of its therapeutic pipeline.
The investment round also saw participation from RTW Investments, Nextech Invest, and other new investors, alongside existing backers such as ARCH Venture Partners, Forbion Ventures, and ND Capital. The funding will help advance AIR-001, the company’s RNA editing candidate for AATD, into Phase 1/2 clinical trials.
“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 strong support from new and existing investors enables us to accelerate the development of AIR-001 for AATD and expand our pipeline of RNA editing therapies.”
AATD is a genetic disorder caused by mutations in the SERPINA1 gene, leading to a deficiency of functional alpha-1 antitrypsin (M-AAT) protein and resulting in lung and liver disease. AIR-001 is designed to precisely correct the most common mutation (PiZ) and restore M-AAT production, potentially offering a safer and more effective treatment option.
“Patients with AATD have limited treatment options, and AIRNA’s RNA editing approach has the potential to deliver a best-in-class therapy,” said Ken Greenberg, M.D., partner at Venrock Healthcare Capital Partners. “We are excited to support AIRNA in its mission to bring novel RNA-based treatments to patients in need.”
AIRNA’s proprietary RNA editing platform leverages natural cellular mechanisms to correct disease-causing mutations and introduce beneficial genetic variations. The company is also developing therapies for cardiometabolic and other diseases, with the goal of transforming patient care through precision genetic medicine.
As part of the funding round, AIRNA announced the addition of Melissa McCracken, Ph.D., a partner at Nextech Invest, to its Board of Directors. Dr. McCracken brings expertise in building innovative biopharma companies with a focus on precision medicine.
With plans to submit a clinical trial application for AIR-001 in the second half of 2025, AIRNA is poised to advance its pipeline of RNA editing therapeutics, potentially reshaping the treatment landscape for genetic diseases.
