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Substituting a hydrogen atom with its heavy isotope deuterium may improve the pharmacokinetic and/or toxicity profile of a drug compared with its non-deuterated counterpart. This article highlights milestones in the field of deuteration in drug discovery and development, and discusses recent examples of its application, which have shifted towards deuteration of novel drug candidates instead of developing deuterated analogues of marketed drugs.
The low number of clinical trials in Africa and biomedical datasets that disproportionately represent populations of European ancestry contribute to the suboptimal efficacy and safety of some medicines in African populations. To address these disparities, we propose greater incorporation of African data into drug discovery and development, as well as the development of African-centric preclinical and clinical models and tools.
GSK’s Arexvy and Pfizer’s Abrysvo provide older adults with a much-needed vaccine for respiratory syncytial virus (RSV) — and raise hopes for an option for infants too.
Many diseases involve reduced or absent levels of a particular protein and would benefit from therapies that increase gene expression. In their Review, Khorkova et al. discuss the growing range of RNA-targeted therapies in development that aim to boost gene expression, including nucleic acid-based therapeutics targeting the complex regulatory network of non-coding RNA species.
The FDA approved tofersen for amyotrophic lateral sclerosis based on the drug’s ability to lower blood levels of neurofilament light (NfL) — establishing a proof of potential for this neuroscience biomarker that could have implications for other diseases of the brain.
Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS), discusses the centre’s audacious goal to have 25% of diseases covered by the experimental drug pipeline by 2032.
The COVID-19 pandemic spurred a wave of rapid and collaborative drug discovery efforts. This Perspective article summarizes scientific drivers and considerations behind such antiviral small-molecule discovery programmes and proposes strategies to accelerate future efforts.
