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Financing led by Venrock Healthcare Capital Partners with co-lead Forbion Growth, and participation from RTW Investments, Nextech Invest, and other new investors Current investors ARCH Venture Partners, Forbion Ventures, ND Capital, and others also participated AIRNA expected to file clinical trial application for Alpha-1 antitrypsin deficiency (AATD) RNA editing product candidate (AIR-001) with potential best-in-class profile in 2H 2025 CAMBRIDGE, MA, USA & TÜBINGEN, Germany I April 01, 2025 I AIRNA, a biotech company pioneering RNA editing therapeutics to transform the lives of patients with rare and common conditions, today announced the closing of an oversubscribed $155 million Series B financing. The financing was led by Venrock Healthcare Capital Partners, co-led by Forbion Growth, and included participation from RTW Investments, Nextech Invest, ARCH Venture Partners, Forbion Ventures, ND Capital, and other new and existing investors. AIRNA will use the proceeds to advance its lead RNA editing product candidate (AIR-001) for AATD into a Phase 1/2 clinical trial, and to develop a pipeline of novel RNA-editing medicines for cardiometabolic and other diseases. “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 Chief Executive Officer 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.” 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. “Patients with AATD have limited therapeutic options, and AIRNA is developing a potentially best-in-class medicine that has been optimized for potency, durability, and safety,” said Ken Greenberg, M.D., partner at Venrock Healthcare Capital Partners. “Venrock is thrilled to collaborate with the tremendous team at AIRNA, and a world-class investor syndicate, to bring novel RNA editing medicines to patients.” 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 identifies variants that drive health through human genetics, and is developing new medicines to transform the lives of patients with cardiometabolic and other diseases. “AIRNA’s innovative approach to RNA editing has the distinctive potential to improve health across large populations by introducing healthy genetic variants for many conditions. We are excited to support the expansion of AIRNA’s pipeline of life-changing medicines and the advancement AIR-001 into the clinic,” said Dirk Kersten, Managing Partner at Forbion. Alongside this financing, AIRNA announced the appointment of Melissa McCracken, Ph.D., a partner at Nextech Invest, to the Board of Directors. Dr. McCracken has a track record of growing the next generation of innovative biopharma companies, with a focus on precision approaches. About AIRNA AIRNA is harnessing advances in genetics to develop transformative RNA-editing medicines that improve human health across both rare and broad populations. RNA editing offers the precision of genetic medicine approaches while maintaining treatment convenience, flexibility, and reversibility. Our proprietary platform is based on groundbreaking research by pioneers of the field and company co-founders Thorsten Stafforst (University of Tübingen) and Jin Billy Li (Stanford University) and enables optimal potency, safety, and delivery. AIRNA is advancing a robust pipeline of therapeutic candidates that are designed to provide functional cures for severe or chronic diseases by repairing harmful genetic variants or introducing beneficial variants promote optimal health. AIRNA’s lead program has the potential to be a best-in-class therapeutic for alpha-1 antitrypsin deficiency (AATD). AIRNA has headquarters in Cambridge, MA, with research operations in Tübingen, Germany. Learn more at https://airna.com/ . SOURCE: AIRNA

AIRNA’s total capital raised so far is $245m. Credit: sesame via Getty Images. AIRNA has raised $155m in Series B financing to advance its RNA editing pipeline, and kick-start a clinical trial for its lead candidate, AIR-001. Venrock Healthcare Capital Partners and Forbion Growth co-led the new round, bringing AIRNA’s total raised capital to $245m. The US and Germany-based biotech plans to file a clinical trial application (CTA) for its lead candidate, AIR-001, for alpha-1 antitrypsin deficiency (AATD) by H2 2025. The company is developing AIR-001 as an investigational RNA editor – a therapy designed to modify RNA sequences to correct disease-causing mutations. This approach is different to DNA editing approaches such as clustered regularly interspaced short palindromic repeats (CRISPR), which make permanent changes to the genome. RNA editing is a reversible process that allows for targeted corrections without altering the underlying genetic code. The Series B funds will support the launch of a Phase I/II clinical trial for AIR-001 and the further development of other RNA-editing candidates, primarily targeting cardiometabolic diseases. AATD is a rare genetic disorder caused by mutations in the SERPINA1 gene, which encodes the AAT protein. Under normal circumstances, AAT is produced in the liver and transported to the lungs, where it regulates enzyme activity to prevent tissue damage. In patients with AATD, mutations can prevent AAT from exiting the liver, leading to toxic protein accumulation that damages the liver while also depriving the lungs of necessary protection. This can result in progressive lung disease, liver dysfunction, and symptoms such as shortness of breath, fatigue, and jaundice. The AATD treatment landscape is becoming increasingly competitive as multiple biotechs advance RNA-editing candidates. Korro Bio is investigating its candidate KRRO-110 in the Phase I/IIa REWRITE clinical trial (NCT06677307), with interim data expected in H2 2025. The company announced last month that the dosing of the first two cohorts had been completed, and the trial is expected to conclude next year. Wave Life Sciences is developing WVE-006, which demonstrated targeted RNA editing in the first two patients treated in the Phase Ib/IIa RestorAATion-2 clinical trial (NCT06405633). The growing interest in RNA editing has also drawn investment from major pharmaceutical companies. In November 2023, Roche expanded its partnership to develop a one-time RNA editing therapy using ShapeTX’s AI-powered platform for an undisclosed disease. In September 2024, Novo Nordisk and Korro Bio announced a collaboration leveraging Korro’s OPERA platform to enable oligonucleotide-directed RNA edits for two undisclosed targets, initially focused on cardiometabolic diseases. The deal included up to $530m in upfront, development, and commercial milestone payments, along with tiered royalties and R&D funding. Not all genetic medicine approaches for AATD have progressed as planned. In January 2025, Intellia Therapeutics discontinued the development of its CRISPR-based gene therapy NTLA-3001, which was intended to correct the SERPINA1 mutation at the DNA level. Preparations for the Phase I/II trial were halted, along with other early-stage programmes, signalling a shift in the company’s pipeline priorities. With fresh funding and a CTA planned for later this year, AIRNA is positioning itself within the growing RNA editing field. Forbion managing partner Dirk Kersten said: “AIRNA’s innovative approach to RNA editing has the distinctive potential to improve health across large populations by introducing healthy genetic variants for many conditions. We are excited to support the expansion of AIRNA’s pipeline of life-changing medicines and the advancement of AIR-001 into the clinic.”