Regulation and new intellectual property laws are needed to reduce the cost of gene-editing treatments and fulfil their promise to improve human health.
Even in wealthy countries, health-care systems are ill-equipped to shoulder the high initial costs associated with gene therapies. In 2021, therapeutics developer Bluebird Bio in Somerville, Massachusetts, withdrew plans to market a gene therapy for β-thalassaemia — another blood disorder — in Europe, after failing to reach an agreement with European authorities over the price.
Low- and middle-income countries, meanwhile, are left entirely in the lurch. This is especially painful given that some of the diseases under consideration, such as β-thalassaemia and sickle-cell disease, are more common in poorer parts of the world than in wealthy nations. In some sub-Saharan regions, for example, it is estimated that about 2% of children are born with sickle-cell disease. This is likely to be an underestimate, given how little screening is taking place.
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