One of the latest entrants to the RNA editing field has tripled its funding and plans to start a clinical trial in a rare liver disease as soon as next year.
AIRNA, which launched with $30 million last September, has added $60 million to its Series A financing, the company announced Wednesday. Its first program is focused on making a single base edit in short-lived RNA molecules to treat alpha-1 antitrypsin deficiency, also known as AATD.
AATD, a genetic condition that affects the liver and lungs, has emerged as a favorite first disease for biotech companies in which to test new genetic medicines.
Two other companies developing RNA editing therapies for AATD are ahead of AIRNA. Wave Life Sciences began a clinical trial last year. And later this year, Korro Bio expects to ask regulators for permission to begin its own study. But AIRNA CEO Kris Elverum told Endpoints News that he isn’t worried about being the third entrant.
“What we are focused on is not being first in patients but being best for patients, and developing a medicine that can be preferred by patients for the long term,” Elverum said. Part of the benefit could come from convenient and infrequent dosing, he added.
While many companies developing in vivo gene editing therapies are using an infusion of lipid nanoparticles to get the therapy into the liver, Elverum said his company’s AATD therapy will be a subcutaneous injection based on a proprietary GalNAc conjugate technology. Alnylam Pharmaceuticals also uses GalNAc conjugates to shuttle its gene-silencing RNAi medicines to the liver.
Forbion, a European life science-focused venture capital firm, led the new funding. New investors including Ono Venture Investment and Alexandria Venture Investments, and existing investors including ARCH Venture Partners, also contributed.