AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.
RNA editing startup AIRNA closed its series B financing round on Tuesday, counting $155 million in earnings that will allow it to perform clinical trials on its lead molecule.
The Cambridge, Mass. and Tübingen, Germany–based company is developing an investigational RNA editor called AIR-001 to treat alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder that commonly affects the lungs and liver. Tuesday’s raise will help the biotech launch a Phase I/II clinical trial for AIR-001 and further develop its pipeline of other RNA-editing candidates to primarily target cardiometabolic diseases.
AATD is caused by mutations in the SERPINA1 gene, which encodes the AAT protein. Under healthy circumstances, AAT is produced in the liver and travels to other organs, including the lungs, where it helps moderate the activity of enzymes that would otherwise cause tissue damage. Mutations in the AAT protein can prevent it from leaving the liver, causing damaging build-up there and preventing it from performing its protective actions in the lungs. Common symptoms of AATD include shortness of breath, extreme fatigue and jaundice.
AIRNA’s AIR-001 addresses AATD by targeting and repairing the most common pathologic SERPINA1 mutation in the RNA transcript, restoring the expression of functional AAT protein. As per the biotech’s website, AIR-001 “aims to provide a functional cure” for AATD by “repairing the disease-driving genetic variant” underlying the disease. AIRNA has completed in vivo proof-of-concept studies for AIR-001.
AIRNA’s series B was co-led by Venrock Healthcare Capital Partners and Forbion Growth. Institutional funders such as ARCH Venture Partners, RTW Investments and Nextech Invest also participated.
With its raise on Tuesday, AIRNA follows in the footsteps of other biotechs that have successfully drummed up investor support even as macro-level concerns plague the industry.
Last week, for instance, Hillstar Bio entered the autoimmune arena with $67 million in series A capital, which it will use to advance a pipeline of molecules that can selectively deplete pathogenic cells while leaving healthy ones untouched. A day earlier, Temper Bio brought in $70 million in series B funds to advance its candidates for substance-use disorders.
Late last month, Eikon Therapeutics announced that its series D financing round had resulted in an initial haul of $350.7 million, which will go toward the development of an investigational activator of toll-like receptors 7 and 8 for the treatment of melanoma. Eikon CEO Roger Perlmutter at the time said that he expects more investors to continue pitching in.
