LITTLE more than a decade ago, a new technology was revealed to the world: CRISPR, a way to change DNA inside cells in a more precise way than ever before. The news triggered a storm of media coverage and earned its developers a Nobel prize in 2020. This year, the first genetic therapy based on CRISPR is set to be approved.
This is for sickle cell disease, a condition in which a faulty protein causes red blood cells to form an abnormal 鈥渟ickle鈥� shape, which can lead to blocked blood vessels, agonising pain and even organ damage. The CRISPR approach activates a gene that can make the correctly-shaped blood cells. This one-off treatment can relieve a lifetime of hardship. Yet there is a catch. Although a price for the therapy, made by Vertex Pharmaceuticals, hasn鈥檛 been announced, analysts predict it could be between $1 million and $3 million per person.
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Sickle cell disease is one of the most common conditions to have been successfully targeted by a genetic therapy and the treatment therefore has a huge potential market. But of the approximately 100,000 people with the condition in the US, where Vertex Pharmaceuticals is based, many are on publicly-funded Medicaid. That set-up can鈥檛 afford this treatment. It would be a tall order for any healthcare system in the world to be able to offer it to all those who could benefit. And this is just one of many CRISPR therapies in development (see 鈥淗ow CRISPR therapy could cure everything from cancer to infertility鈥�).
It is unclear how authorities will react. There are solutions, including streamlining regulatory processes to make it cheaper to test the efficacy and safety of these therapies, as well as the promise of 鈥渙ff-the-shelf鈥� CRISPR treatments that will significantly reduce costs. But until then, celebrations over CRISPR鈥檚 incredible clinical potential need to be tempered with the knowledge of its limitations. We don鈥檛 just need to figure out how to create new medicines 鈥� but also how to get them to everyone who needs them.
