AAV9-corsATP7A

AAV9-corsATP7A is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy designed for the treatment of Menkes disease and related ATP7A deficiency disorders. The vector carries a codon-optimized, engineered human ATP7A transgene (corsATP7A) that has been shortened from 4.5kb to 3.12kb by retaining only two of the six copper-binding domains. This design optimization enhances AAV encapsidation, yield, and quality while maintaining functional copper transport activity. The therapy aims to restore copper homeostasis by providing a functional version of the ATP7A protein, which is deficient in Menkes disease patients. It is currently in preclinical development, with research focusing on establishing potency assays for IND approval.