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Unlocking the potential of mRNA for the future treatment of rare diseases 

Drug Discovery World

Moderna Therapeutics, a biotechnology company that has developed SARS-CoV-2 mRNA vaccines, has at least six rare disease programmes currently in preclinical and clinical development (Table 1). At this stage, qPCR can be used to check for IVT efficiency, residual DNA contamination and adventitious agents such as microbes.