Kygevvi: animal efficacy model or human cells?

“Primary pharmacodynamics A total of five non-GLP primary pharmacodynamic studies, including three literature studies, were conducted using wild-type (WT), Tk2KO, and Tk2KI mouse models to evaluate the effects of nucleoside and nucleotide therapies on TK2 deficiency (TK2d).”

“Both models exhibit mitochondrial dysfunction and respiratory chain defects, mimicking the severe mitochondrial DNA (mtDNA) depletion caused by thymidine kinase 2 deficiency, which closely resembles human infantile encephalomyopathy.”

“the targeted mechanism of action of doxecitine and doxribtimine, and treatment of TK2 deficient animal models that provides additional mechanistic support and evidence of a survival benefit.”