Luis A. Brito1, Michelle Chan1, Armin Hekele2, Christine A. Shaw1, Tom Carsillo1, Anja Seubert3, Gillis R. Otten1, Clayton W. Beard2, Antu K. Dey2, Anders Lilja1, Christian W. Mandl1, Susan W. Barnett1, Philip R. Dormizer1, Jeffrey B. Ulmer1, Manmohan Singh2, Derek T. O’Hagan1, Andrew J. Geall1
1Novartis Vaccines and Diagnostics, Cambridge, MA USA
2Novartis Vaccines and Diagnostics, Holly Springs, NC USA
3Novartis Vaccines and Diagnostics, Siena, Italy
We have reinvented the gene vaccine by creating a synthetic self-replicating RNA vaccine platform capable of providing a rapid immune response to prevent and treat current and emerging infectious threats. We have achieved proof of concept for 1st generation self-replicating RNA vaccines in small and large animal models. The vaccine RNA is produced by an enzymatic transcription reaction and formulated with non-viral delivery systems, thereby avoiding the limitations of cell culture production that complicate production of other vectored delivery systems. Given the many positive attributes of nucleic acid vaccines, our results suggest that a comprehensive evaluation of non-viral technologies to deliver self-amplifying RNA vaccines is warranted.