Evidence for a fluid clearance pathway in the central nervous system known as the glymphatic system has grown in the past decade. Nedergaard and colleagues overview the evidence for the glymphatic system and its role in disease, and discuss opportunities to harness the glymphatic system therapeutically; for example, by improving the effectiveness of intrathecally delivered drugs.

The potential of therapeutically targeting RNA structures with small molecules is being increasingly recognized. Here, Disney and colleagues review strategies to identify, validate and optimize small-molecule RNA binders. Examples of existing RNA-targeted small molecules, as well as challenges and future directions in the field, are discussed.

Genome-editing drugs that make precise corrections to DNA are entering clinical trials for cardiovascular and haematological diseases.

Antibody function is dependent on avidity — the accumulated strength of multiple affinity interactions between the antibody, antigen, cell surface receptors and other antibodies. In this Review, Oostindie et al. discuss the role of avidity in eliciting antibody functional responses and review the current engineering strategies for manipulating avidity interactions in antibody-based therapies.

The UK Biobank houses an extensive collection of biological samples and health information from 500,000 volunteers, providing clues to the genetic and environmental drivers of disease. Naomi Allen, the project’s chief scientist and an epidemiologist at Oxford University, discusses the exploding body of findings from this 20-year-old biomedical resource and the prospects for the expansion of this biopharmaceutical treasure trove.