TS-005572 — This novel gene replacement strategy uses an adeno-associated viral vector to treat Cockayne syndrome type B (CSB). CSB is a rare neurodegenerative disorder caused by mutations in the ERCC6 or ERCC8 genes, both of which are responsible for DNA repair, causing tremors, ataxia, seizures, and cachectic dwarfism, significantly impacting their quality of life. An AAV-based gene replacement therapy will effectively combat this disorder by delivering a functional copy of the defective gene into target cells, thereby restoring normal gene expression.