AAVDJ-ERCC6

AAVDJ-ERCC6 (also referred to as AAVDJ-CP040-coERCC6) is an experimental adeno-associated virus (AAV) vector-based gene therapy designed for the treatment of Cockayne syndrome B (CSB). CSB is a rare, inherited DNA repair disorder caused by pathogenic variants in the excision repair cross complementation group 6 (ERCC6) gene, leading to accelerated aging and severe neurodegenerative complications. The therapy utilizes a synthetic AAVDJ capsid to deliver a human codon-optimized version of the ERCC6 gene (coERCC6) under the control of the CP040 mini-promoter. Preclinical research conducted at the University of Minnesota has shown that intravenous administration of AAVDJ-ERCC6 in mouse models reduces oxidative and genomic stress, restores CSB protein expression in key brain regions (cortex, hippocampus, and thalamus), and improves behavioral outcomes. The program is currently in preclinical development with the goal of advancing into human clinical trials.