Self-amplifying mRNA (SAM) is a next generation vaccine platform technology that is undergoing the early-stage development in GSK Vaccines. SAM vaccine platform is based on a synthetic, self-amplifying mRNA that encodes the alphavirus RNA amplification machinery and the vaccine antigen. This innovative vaccine platform is capable of eliciting both humoral and cellular immune responses. In preclinical studies, SAM-based vaccines demonstrate substantially enhanced potency, by producing multiple copies of vaccine RNA templates <i>in vivo </i>and by self-adjuvanting activity of amplifying RNA. The production of mRNA utilizes a cell-free enzymatic transcription reaction that can be completed in a few hours. This fast production allows scaling up in manufacturing capacity and enables a rapid response to pandemic situations.<br/>An mRNA vaccine, delivered with a nanoparticle-based delivery system, is complex in nature and must overcome scientific, technical, and regulatory challenges. In order to build a proper control strategy during CMC development for mRNA-based vaccines and therapeutics, it is prerequisite to establish analytical methods to support novel process development and drug product characterization. This presentation will discuss using multiple physicochemical, molecular biology, and in vitro potency methods to characterize critical quality attributes (CQAs) for a successful early CMC development of SAM based vaccines.<br/>

Characterization of critical quality attributes of self-amplifying mRNA: A novel vaccine/therapeutic modality

2019 Spring National Meeting

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