CERC-913

CERC-913 is a preclinical-stage ProTide nucleotide therapy designed as a substrate replacement for the treatment of mitochondrial DNA depletion syndrome (MDS), specifically targeting deoxyguanosine kinase (DGUOK) deficiency. Acquired by Cerecor (now Avalo Therapeutics) through its acquisition of Ichorion Therapeutics in 2018, CERC-913 utilizes the ProTide phosphoramidate prodrug technology to deliver nucleotide precursors that bypass the metabolic block caused by DGUOK mutations. By restoring the mitochondrial deoxyguanosine triphosphate (dGTP) pool, the therapy aims to maintain mitochondrial DNA (mtDNA) copy numbers and prevent the severe hepatocerebral symptoms associated with the disorder. The program was positioned for development via the FDA's 505(b)(2) regulatory pathway to leverage existing safety data and accelerate clinical entry.