rAAV9-ERCC6

rAAV9-ERCC6 is a gene therapy candidate designed for the treatment of Cockayne Syndrome complementation group B (CS-B), an ultra-rare autosomal recessive progeroid disorder. The therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) vector to deliver a functional copy of the ERCC6 (also known as CSB) gene. ERCC6 encodes a protein essential for DNA damage repair, transcriptional regulation, and mitochondrial homeostasis. By restoring ERCC6 expression, the therapy aims to address the multisystemic symptoms of CS-B, including neurological abnormalities and growth deficits. The rAAV9 serotype is specifically chosen for its ability to cross the blood-brain barrier, enabling systemic delivery to the central nervous system. Development is being led by researchers at the Universidade do Algarve and UMass Chan Medical School.

Other names

rAAV9-CSBrAAV-9-CSBrAAV 9-CSB