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It's been barely a decade since two researchers came up with a plan to edit DNA in order to disrupt genetic disorders. But the UK has just become the first country to approve a therapy based on Crispr gene editing, authorising a treatment for sickle cell disease and beta thalassemia. DNA acts as an instruction manual for the body. Crispr technology works like a molecular set of scissors that allow scientists to make targeted edits in DNA, changing genetic code at a precise point in the genome. So far, about 70 patients have been successfully treated using the disruptive gene editing technology, curing them of debilitating blood disorders such as sickle cell disease.
The two biotech companies at the forefront of developing the therapy are CRISPR Therapeutics and Vertex Pharmaceuticals. Over 100 clinical trials are under to use Crispr to treat both rare and prevalent diseases, such as blood disorders, as well as cancers, diabetes, blindness, and HIV. One pharma company has even started trials using Crispr technology on a patient with a genetic condition that causes high cholesterol, a therapy, which could one day be marketed to millions of patients.
But the procedure is complex. Patients undergo costly and risky chemotherapy to wipe out their blood cell stems in the bone marrow and make room for cells altered outside the body. And the price tag could be more than $1mn per person, beyond the reach of many. For example, over half of the more than 300,000 people born with sickle cell disease each year live in countries where few people could afford treatment; Nigeria, the Democratic Republic of Congo, and India.
Analysts say the invention of Crispr is to medicine what the invention of the transistor was to computer design and that drug makers are already showing market interest in Crispr companies. Big pharma groups are sitting on huge cash piles and a number of them need to build up their drug pipelines before significant patterns expire at the end of the decade. Despite all the early success, though, the questions around Crispr, such as the long-term effects on patients, how much it will cost, and who will have access to it, still need answers.
