hATP7B mRNA-LNP

hATP7B mRNA-LNP is an mRNA-based therapeutic candidate developed by Innorna for the treatment of Wilson Disease (WD). It consists of a nucleoside-modified, codon-optimized human ATP7B mRNA encapsulated within a proprietary, liver-tropic lipid nanoparticle (LNP). Wilson Disease is an autosomal recessive disorder caused by mutations in the ATP7B gene, which encodes a P-type copper transporter primarily expressed in hepatocytes. The loss of ATP7B function leads to toxic copper accumulation in the liver and other organs, such as the brain and eyes, resulting in hepatic, neurological, and ophthalmologic complications. By delivering the hATP7B mRNA to hepatocytes, the therapy aims to restore functional ATP7B protein expression, thereby normalizing copper transport and hepatobiliary copper elimination. Preclinical studies in *Atp7b−/−* mice have demonstrated that the treatment can restore serum ceruloplasmin activity, reduce hepatic copper burden, lower urinary copper, and increase fecal copper excretion.

Other names

hATP7B-mRNA-LNPhATP-7B-mRNA-LNPhATP 7B-mRNA-LNP