The gene-therapy revolution risks stalling if we don’t talk about drug pricing

“We wish to suggest a structure for the salt of deoxyribose nucleic acid (D.N.A.),” wrote James Watson and Francis Crick in this journal in 1953 (J. D. Watson and F. H. C. Crick Nature 171, 737–738; 1953). “This structure has novel features which are of considerable biological interest.”

In the 70 years since those famous words were published, researchers have poured huge effort into unravelling those features and harnessing them for medicine. The result is a flourishing understanding of the genetic causes of diseases — and a host of therapies designed to treat them.

Seventy years from now, the world might look back on 2023 as a landmark, as well. This year could see the first authorization of a therapy based on CRISPR–Cas9 gene editing, that involves tweaking the DNA in the body’s non-reproductive (somatic) cells. Gene editing allows scientists — and could soon permit clinicians — to make changes to targeted regions in the genome, potentially ‘correcting’ genes that cause disease. Regulators in the United States, the European Union and the United Kingdom are evaluating...