AAV8-FGF19 Variant M70

AAV8-FGF19 Variant M70 is an AAV8-based gene therapy delivering the cDNA of FGF19 variant M70, an engineered fibroblast growth factor 19 analog designed to retain bile acid–regulatory and metabolic benefits while eliminating FGF19-associated hepatocarcinogenic signaling. M70 contains three N‑terminal substitutions (A30S, G31S, H33L) and a five–amino acid deletion, acting as a biased agonist of FGFR4/β‑Klotho that suppresses CYP7A1 and bile acid synthesis, improves lipid and glucose metabolism, and mitigates NASH features in mouse models, without inducing liver tumors seen with wild-type FGF19 under chronic overexpression. Developed for nonalcoholic steatohepatitis and other liver metabolic disorders; preclinical development reported by Gordian Biotechnology.[2][1][5][3]

Other names

M70M-70M 70engineered FGF19 M70non-tumorigenic FGF19 variant M70