REGENX’s NAV® rAAV Vector Technology is ideally suited for applications where long-term, stable gene expression is required. However, some therapeutic genes to be delivered must be precisely controlled in terms of levels and/or timing of expression – for example, if expressing a therapeutic cytokine or monoclonal antibody. Furthermore, for certain therapeutics, the safety profile would be significantly improved by the ability to withdraw the therapy. For these conditions, REGENX has exclusive rights to NAV Regulation Technology for in vivo gene delivery mediated by viral vectors.
NAV Regulation is built from human proteins to minimize the potential for immunogenicity. Therapeutic transgene expression is initiated and levels controlled by administration of an orally bioavailable rapamycin analog that is non-immunosuppressive. This technology will be used for therapeutics under development at REGENX, and may be sublicensed to partners.