Drug notes:
Also RD undisclosed
About:
AIRNA Bio is developing oligonucleotide-based therapeutics that mediate RNA editing for patients with rare and common diseases. AIRNA uses a unique oligonucleotide drug construct to bind to a specific RNA sequence and then attract endogenous ADAR enzymes to the RNA. ADAR enzymes can mediate the chemical transformation of a specific adenosine (A) to inosine (I), which is read as guanosine (G) when translating RNA to protein. This A-to-I edit can fix pathogenic point mutations, induce therapeutically-effective gain-of-function or loss-of-function mutations, or change protein conformation.