Pharmaceutical Technology

SEPTEMBER 30, 2022

Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. million for the transfusions themselves and $3.7 bluebird has opted to launch it at a cost of $2.8

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Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. Sickle cell disease is an inherited blood disorder that is caused by mutations in the HBB gene, which codes for the oxygen-carrying protein haemoglobin in red blood cells.

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