CURE CLCN6 INC

Description

Cure CLCN6 was formed in February 2024, and is the only patient advocacy group in the world for mutations on the CLCN6 gene. Mutations on the CLCN6 gene are rare and neurodegenerative. Cure CLCN6 is working with UMass Chan Medical School to develop a novel gene therapy to treat this mutation, that will not only save the lives of affected children, but significantly improve the quality of life for them and their families. The mouse model to better understand these mutations, and to test the safety and efficacy of the gene therapy is being completed by the Jackson Laboratory. The gene therapy is on schedule to be ready for a clinical trial in 2027.