CLN8 gene therapy

CLN8 gene therapy is an investigational **AAV9-based gene replacement therapy** for **CLN8 disease**, a rare neuronal ceroid lipofuscinosis form of Batten disease caused by biallelic pathogenic variants in **CLN8**. The therapy uses a **self-complementary adeno-associated virus serotype 9 vector** to deliver a functional human **CLN8** transgene to the central nervous system with the goal of restoring CLN8 protein expression and improving lysosomal biology. Based on the provided context, the program has been developed in collaboration with **Amicus Therapeutics** and has shown strong preclinical activity after **intracerebroventricular** administration in CLN8 disease mouse models, including broad CNS transgene expression, reduced disease pathology, improved behavioral phenotypes, and marked lifespan extension.

Other names

CLN8 gene therapyCLN-8 gene therapyCLN 8 gene therapyAAV9 CLN8 gene therapyAAV-9 CLN8 gene therapyAAV 9 CLN8 gene therapyscAAV9 CLN8 gene therapyscAAV-9 CLN8 gene therapyscAAV 9 CLN8 gene therapy