Perspectives on RNA therapeutics, translational development and biotechnology R&D, drawing on experience across discovery, preclinical development, clinical translation, CMC and programme strategy.
Choosing the first human dose for an mRNA therapeutic
Selecting a first-in-human dose for an mRNA therapeutic requires more than scaling an animal dose. Delivery, translation, encoded-protein pharmacology, safety and biomarkers all contribute to defining a safe and informative starting point.
PK/PD for mRNA therapeutics: what should count as exposure?
For mRNA therapeutics, the easiest exposure to measure may not be the one that drives pharmacology. This article examines how distribution, productive intracellular delivery, encoded-protein exposure and downstream response should be connected.