AAVrh.10CUhCLN2

AAVrh.10CUhCLN2 is an investigational gene therapy designed to treat late infantile neuronal ceroid lipofuscinosis (LINCL), also known as late infantile Batten disease, a lysosomal storage disorder caused by mutations in the CLN2 gene. The therapy uses an adeno-associated virus serotype rh.10 (AAVrh.10) vector to deliver a functional copy of the human CLN2 gene directly into the brain, aiming to restore TPP1 enzyme activity and slow or halt neurodegeneration characteristic of LINCL[1][3][5]. The vector is derived from a primate source and has demonstrated safety and efficacy in preclinical models; it is considered more effective than earlier vectors such as AAV2 for CNS delivery[5]. The primary indication is for children with confirmed LINCL/Batten disease.

Other names

AAVrh.10-hCLN2AAVrh.10-CLN2