Company’s ‘precise and reversible’ RNA-editing approach is designed to tackle chronic diseases in large populations.
Genetic medicine developer AIRNA has secured a $155 million Series B financing to advance its portfolio of RNA editing therapeutics. The funding will primarily support the development of its lead candidate, which targets a genetic mutation linked to liver and lung disease, while also expanding its focus on cardiometabolic diseases and other chronic conditions.
Headquartered in Cambridge, MA, with research operations in Tübingen, Germany, AIRNA’s editing platform is based on the ADAR enzyme, which facilitates precise edits in RNA molecules. Unlike DNA-editing technologies that create permanent genomic modifications, the company says its approach enables therapeutic interventions that are both precise and reversible – a critical feature for management of age-related and chronic diseases, where long-term therapeutic flexibility is essential.
The AIRNA platform uses chemically optimized guide RNAs to direct ADAR to specific RNA targets, minimizing unintended edits and maximizing therapeutic potential. The company’s strategy aims to either correct harmful genetic mutations or introduce beneficial variations that enhance health outcomes in the population – an interesting proposition from a longevity perspective.
AIRNA says it is leveraging its RNA-editing capabilities to address cardiometabolic diseases and other chronic conditions. By identifying genetic variants associated with improved health outcomes, such as those conferring resilience to cardiovascular disease, the company seeks to replicate these effects through targeted RNA edits.
The initial focus of AIRNA’s clinical program is a condition called AATD, which arises from mutations in the SERPINA1 gene, leading to a deficiency of a key protein called alpha-1 antitrypsin (M-AAT). Although often categorized as a rare disease, AATD is a common genetic disorder with a wide range of clinical manifestations, including lung and liver disease. AIRNA’s lead therapeutic is designed to repair the RNA transcript affected by the most prevalent AATD-related mutation, thereby restoring normal M-AAT production.
“AIRNA is developing a new class of genetic medicines that could provide functional cures for a wide range of diseases,” said AIRNA CEO Kris Elverum. “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.”
The recent funding round, led by Venrock Healthcare Capital Partners and Forbion Growth, alongside a host of other investors, will support AIRNA’s lead program into Phase 1/2 clinical trials, while further expanding its therapeutic portfolio.
“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,” said Forbion’s Dirk Kersten.
Photograph: Edovideo/Envato
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