AIRNA

AIRNA is a biotechnology company pioneering RNA editing therapeutics to transform the lives of patients with rare and common diseases. The company RESTORE+ platform enables the development of novel medicines with the potential to deliver functional cures by precisely editing RNA to correct disease-causing mutations. AIRNA lead therapeutic candidate, AIR-001, targets alpha-1 antitrypsin deficiency (AATD), an inherited genetic disease, and has demonstrated best-in-class potential achieving up to 59% precise RNA editing in vivo and restoring 40 uM functional M-AAT in a mouse model of AATD. Beyond AATD, AIRNA is advancing cardiometabolic RNA-editing programs that introduce protective variants for cardiovascular disease and drive reduction of LDL cholesterol levels, expanding the therapeutic potential of RNA editing beyond rare diseases to common conditions. The company platform technology can deliver individual therapeutic solutions at scale, opening possibilities for treating a wide range of genetic diseases. AIRNA operates with operations in Cambridge, Massachusetts, and Tubingen, Germany, and has appointed industry veteran Todd Bazemore as President and CEO to drive the next stage of company growth, bringing over 30 years of experience leading clinical and commercial-stage biotechs. Founded in 2021 and headquartered in Cambridge, Massachusetts, AIRNA has raised approximately $245 million in Series B funding from investors including Arch Venture Partners, ND Capital, Novalis LifeSciences, Forbion, Venrock, RTW Investments, Nextech Invest, Codon Capital, Fast Track Initiative, Ono Venture Investment, and Alexandria. The company employs approximately 84 people.

Rare Diseases > Drugs > Metabolic Disorders,

Genomics > Applied Genomics > Clinical Genomics > Therapeutics

College Wise > Tsinghua University, Harvard University