AIRNA, a biotech startup based in Cambridge, is developing a genetic medicine to treat alpha-1 antitrypsin deficiency (AATD), a rare inherited disorder that leads to liver and lung damage. The company’s therapy aims to edit RNA to address the underlying cause of the disease. While facing competition from other companies, AIRNA received a fresh round of $60 million in funding to support its plans. The therapy recruits an enzyme called ADAR to edit RNA, a process that can repair the mutation causing AATD. AIRNA aims to advance its program to clinical testing in 2025 and expand its drug pipeline to target other diseases amenable to RNA editing.
Source link