AATD is a genetic condition caused by mutations in the SERPINA1 gene, leading to lung and liver disease. AIR-001 aims to precisely repair the most common harmful mutation (PiZ) to restore functional alpha-1 antitrypsin protein production. The funding, led by Venrock Healthcare Capital Partners and Forbion Growth, with participation from other investors, will also support the development of AIRNA’s broader pipeline of RNA-editing medicines targeting cardiometabolic and other diseases.
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. Their 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.
Read the full press release here.
