AAV-ATP7A Gene Therapy Development Pathway
In March 2017, Cyprium and NICHD entered into a worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of a reduced-size, codon-optimized version of the ATP7A gene that is defective in Menkes patients to be used in combination with CUTX-101. AAV-ATP7A gene therapy, which is currently in preclinical development at NICHD, has demonstrated the ability to rescue neurological phenotypes and improve survival when co-administered with copper injections in a mouse model of Menkes disease. Cyprium will work with NICHD to determine the optimal vector design and nominate a candidate for clinical development. The AAV-ATP7A gene therapy has been granted orphan drug designation by the FDA.