DNA Helix
DNA Helix
Image by PublicDomainPictures from Pixabay

This past Thursday, the National Institute for Health and Care Excellence (NICE), published draft guidance recommending the United Kingdom’s National Health Service (NHS) not cover Bluebird Bio’s gene therapy Zynteglo. Zynteglo (betibeglogene autotemcel) “is the first and only one-time gene therapy for transfusion-dependent β-thalassaemia (TDT) that gives patients the potential to achieve transfusion independence.” This revolutionary treatment has the potential to cure patients’ disease and save them from a lifetime of blood transfusions and chronic anemia, among other health issues. The problem is it is EXPENSIVE, costing about ~$1.8M, broken into a 20% down payment and another 20% per year…

Amit Gupta

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