Biotechnology against inherited risk and ageing

Polygenic risk scores discriminate modestly for common diseases — a top-decile score carries perhaps two to three times the average risk for coronary disease or breast cancer — and they perform worse in people of non-European ancestry, because the underlying studies are mostly European. That is the state of the science. The state of practice is a set of large programmes testing whether the scores are useful at the point of care: Our Future Health in the UK is recruiting millions with a view to returning risk information; Genomics England's newborn sequencing study is testing whole-genome screening at birth; Estonia and Finland have returned scores to citizens. What none of these has yet produced is a randomised trial showing that acting on a score changes an outcome. Trials of score-guided screening for prostate and breast cancer are under way. Until they report, the case for population use rests on prediction rather than benefit, and the equity problem is unresolved.

Gene therapy has crossed from promise to product for a few inherited conditions. The first gene-editing treatment, for sickle cell disease and beta thalassaemia, was licensed in 2023; the first therapy designed for a single patient's mutation was given in 2025; and a dozen other single-gene disorders have approved treatments. The population question is not whether they work but for whom and at what price: each is priced in the millions, the conditions are rare, and the long-term follow-up that would show durability and late harm is a few years old at most.

Ageing itself is the furthest out. Drugs that clear senescent cells, and older drugs repurposed against ageing, are in early trials with surrogate endpoints; the trial that would test whether a drug delays several age-related diseases at once has been proposed for a decade and not funded; and the claims made for the GLP-1 drugs as ageing treatments run well ahead of any evidence on lifespan. No intervention has been shown to slow human ageing.

Status

emerging

Direction

strengthening

Would change our view

A randomised trial in which score-guided screening or treatment changed a clinical outcome, or evidence at scale that returning scores caused harm or widened ancestry inequities; a gene therapy priced and delivered at a scale that reaches most eligible patients in a country; or a geroscience trial with a hard clinical endpoint.

Trackers

Gaps

Prediction and calibration in non-European ancestries; implementation evidence outside the UK, US, Estonia and Finland; long-term follow-up of gene therapies; any human trial of an ageing intervention with mortality or disease incidence as the outcome.