CRISPR genome editing has made it possible to cure the blood disorder sickle cell disease, which affects millions of people worldwide. However, most people with the condition are unable to afford the cost of the treatment.
Victoria Gray, born with sickle cell disease, spoke this week at the Third International Summit on Human Genome Editing in London, sharing her experience of the disease and the experimental treatment she received that used the genome editing approach CRISPR/Cas9.
The crippling pain she'd endured since she was a child wasn't really a life, she said, just an existence. 'I felt that I had nothing to lose, I was at the end.'
Four years after the treatment, Gray is now free of symptoms, and is able to 'dream again without limitations,' she said on stage at the summit.
This month, CRISPR Therapeutics and Vertex Pharmaceuticals, who developed the treatment, are expected to file approval paperwork with the US Food and Drug Administration. If approved, it would be the first marketed therapy based on the CRISPR approach.
With an expected price tag of $4-6 million per patient, cost is likely to be a huge barrier for getting treatments to the places in the world where the vast majority of sickle cell patients live, namely countries in sub-Saharan Africa.
'The road from now, having gene editing that is possible and implemented where most people live, it's not that close,' said Professor Ambroise Wonkam, a geneticist at Johns Hopkins School of Medicine, Maryland and the University of Cape Town, South Africa, who also serves as president of the African Society of Human Genetics. 'We have to put that into perspective.'
Read the full article by science writer, Megan Molteni, for STAT.
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