AAV9-ATP7A (Kunming Hope of Health Hospital)

AAV9-ATP7A is an investigational gene therapy developed for the treatment of Menkes syndrome, a rare and fatal X-linked neurodegenerative disorder. The therapy utilizes a recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the ATP7A gene, which encodes a copper-transporting P-type ATPase. Mutations in this gene lead to impaired copper transport across cell membranes, resulting in severe copper deficiency in the brain and other tissues. By restoring functional ATP7A protein expression, the therapy aims to normalize copper homeostasis and prevent the progressive neurological deterioration characteristic of the disease. This investigator-initiated program is being evaluated in an early Phase 1 trial at Kunming Hope of Health Hospital in collaboration with VectorBuilder, Lantu, and 920 Hospital.

Other names

Recombinant adeno-associated virus administrationAAV9-ATP7A gene therapyAAV-9-ATP7A gene therapyAAV 9-ATP7A gene therapyrecombinant adeno-associated virus-Kunming Hope of Health Hospital-Menkes syndrome