OF THE MANY patients who need an organ from a donor, 90% go without. About 240m people live with rare genetic diseases, most of which cannot be treated. Each year poor diets cause more than 10m early deaths. Suffering on such an immense scale can appear hopeless. However, a technique called CRISPR gene editing promises to help deal with these issues and many more—and wise regulation can spur it on.
CRISPR is like an editor that can rewrite DNA letter by letter or gene by gene, to remove harmful mutations or add protective ones. Clinical trials will begin this summer on pig organs edited for transplanting into humans. Last year the first new therapy went on the market. It seemingly cures sickle-cell disease and beta-thalassemia, two blood disorders that afflict millions. If ongoing clinical trials succeed, a one-off therapy could provide lifelong protection against heart attacks. Farming will benefit, too: CRISPR could raise yields or protect crops from climate change. Consumers could soon get white bread with fibre-like starch or tastier varieties of healthy but unpopular foods, such as mustard greens.
But as we report in our Technology Quarterly, now is a critical moment. Since CRISPR’s discovery in 2012, it has begun supplanting old ideas that never reached their potential. Gene therapy, a different technique that uses viruses to insert genes into patients, can treat many rare genetic diseases but is and will remain costly to prepare. Genetically modified (GM) crops, which borrow genes from other species, have faced misguided opposition in Europe and elsewhere. CRISPR offers an alternative to both. But if, unlike them, it is to live up to its promise, it will need to attract a continuing flow of investment—which, in turn, means chalking up some real-life successes.
For that to happen, scientists must show that they can get CRISPR into more types of cells in the body cheaply and easily. The technology would also be boosted if it could serve as a platform to create personalised therapies for people’s individual mutations. That will require new science, but it would also be catalysed by a better system of regulation.
Regulations that govern drugs for rare diseases were not designed for an era of specialist medicines and will hinder patients from receiving new treatments. Developing drugs for a small group of people has always been difficult and many CRISPR companies are struggling, despite government help. But CRISPR is programmable, meaning that the same drug can be tweaked to target many different mutations. On-demand, small-batch drugs for rare diseases could be made more cheaply today if requirements on safety testing and manufacturing standards were loosened. For many desperately ill people who may die before a drug is approved, if it is developed at all, that is a worthwhile trade-off. In America the Food and Drug Administration has already taken some steps towards liberalisation.
Agriculture also badly needs reform. Gene-edited foods fall under GM regulation in many regions, including the European Union, despite being quite different: gene-edited plants have had their own genes tweaked rather than incorporating genes from other species. Mindful of the threat of climate change to food security, Britain is poised to implement new liberal laws governing gene-edited foods; the EU should follow. However, public trust in regulators and scientists could become a problem with the confirmation as health secretary of Robert F. Kennedy junior. He has invested in CRISPR therapies, but is also anti-GM. If America slows down or even goes into reverse, it will be a blow to progress—and humanity. ■
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