Current approaches for in vivo delivery of gene editors require packaging the guide RNA and Cas9 nuclease sequences as DNA within an AAV genome or as mRNA within a lipid nanoparticle (LNP). At Spotlight, we have demonstrated that the much smaller eRNPs can be administered without any packaging. eRNPs have a short half-life in cells, which reduces the risks of off-target editing, immunogenicity, and cellular toxicity as compared with genetically encoded gene editors that may persist for weeks or years.

From a CMC perspective, eRNPs build off decades of experience in biologics manufacturing without any need for viral production or complex encapsulation steps. Therefore, eRNPs can be centrally manufactured and distributed by standard methods for administration at the point of care and significantly broaden the access of gene editing therapeutics.

eRNPs