AAV8-ΔC4ATP7B

AAV8-ΔC4ATP7B is an investigational gene therapy being developed by GeneCradle for the treatment of Wilson's Disease. The therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional, truncated version of the ATP7B gene (the ΔC4ATP7B variant) specifically to the liver. Wilson's Disease is an autosomal recessive disorder caused by mutations in the ATP7B gene, which leads to the toxic accumulation of copper in the liver, brain, and other organs due to impaired biliary copper excretion. By restoring the expression of a functional copper-transporting ATPase, AAV8-ΔC4ATP7B aims to normalize copper metabolism, reduce hepatic copper levels, and reverse liver histopathology. Preclinical studies have demonstrated its ability to normalize serum ceruloplasmin and reduce urinary copper excretion, supporting its progression toward clinical trials.

Other names

GeneCradle Wilson's Disease gene therapyATP7B-AAV8 gene therapyATP-7B-AAV8 gene therapyATP 7B-AAV8 gene therapy