AX-9115

AX-9115 is an early-stage research program developed by ProQR Therapeutics for the treatment of an undisclosed rare metabolic condition. The program utilizes the proprietary Axiomer RNA editing technology, which employs Editing Oligonucleotides (EONs) to recruit the body's endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes. These enzymes catalyze the conversion of Adenosine (A) to Inosine (I) in a target RNA sequence. Because Inosine is recognized as Guanosine (G) by the cellular translation machinery, this A-to-I editing can correct point mutations, modulate protein splicing, or alter protein function. AX-9115 is currently in the discovery phase, and specific details regarding its molecular target or the exact metabolic disease have not been publicly disclosed.