Biotechnology company Airna has successfully secured $155 million in Series B funding to advance its innovative RNA editing treatment platform, with a particular focus on launching clinical trials for alpha-1 antitrypsin deficiency (AATD) later this year.
The company’s leading drug candidate, AIR-001, targets the underlying genetic cause of AATD by correcting defective RNA transcripts, enabling cells to produce properly functioning AAT protein. This approach aims to reduce the risk of lung and liver complications associated with the condition, which affects approximately 100,000 individuals in the United States.
The funding announcement follows recent developments in the RNA editing field, including Wave Life Sciences’ promising Phase 1 results for a similar AATD treatment roughly six months ago. Both companies utilize sugar molecules as delivery vehicles to transport their therapeutic compounds to the liver.
Founded by academic experts Thorsten Stafforst from the University of Tubingen and Jin Billy Li from Stanford University, Airna’s technology harnesses the natural enzyme adenosine deaminase acting on RNA (ADAR) to modify RNA sequences. CEO Kris Elverum emphasizes that AIR-001, administered through subcutaneous injection, offers advantages over gene therapy approaches by providing treatment flexibility without permanent DNA alterations and with potentially lower manufacturing costs.
“We’re focusing on giving patients and their doctors treatment options that could effectively cure the condition while maintaining control over their healthcare journey,” Elverum explained. The company is currently optimizing dosing strategies to maximize the drug’s effectiveness at lower concentrations.
While AATD remains Airna’s primary focus, the company’s technology platform shows promise in other therapeutic areas. Co-founder Stafforst’s research suggests potential applications in
cardiometabolic and neurodegenerative conditions, though specific programs haven’t been publicly disclosed.
The Series B financing round was jointly led by venture capital firms Venrock and Forbion, with participation from RTW Investments, Arch Venture Partners, and Nextech Invest. This latest investment follows Airna’s $90 million raise when it emerged from stealth mode in 2023.
The RNA editing field has attracted significant attention and investment, with companies like Shape Therapeutics, Ascidian Therapeutics, and ADARx Pharmaceuticals pursuing similar approaches. Established biotechnology firms Wave and Korro Bio have also entered the space, while pharmaceutical giants Roche and Novo Nordisk have formed strategic partnerships. However, the long-term success of RNA editing therapeutics in clinical practice remains to be demonstrated.
Commenting on the investment, Forbion managing partner Derk Kersten stated, “The unique potential of Airna’s RNA editing approach lies in its ability to introduce beneficial genetic variants across multiple conditions, potentially improving health outcomes for large patient populations.”
Currently, AATD patients rely on weekly AAT protein injections to manage their condition, as no cure exists. Airna’s approach could potentially offer a more effective treatment option by addressing the underlying genetic cause of the disease.
The company’s development program represents a growing trend in the biotechnology sector, where RNA editing is emerging as a promising therapeutic strategy for genetic disorders. While significant venture capital investment and pharmaceutical industry interest demonstrate confidence in the field, the ultimate success of these approaches will depend on clinical trial results and real-world effectiveness.
The substantial funding secured by Airna reflects investor confidence in both the company’s technology platform and its potential to address unmet medical needs, particularly in rare genetic disorders like AATD.
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