tag stem-cell-therapy 
European Pharmaceutical Review
DECEMBER 27, 2023
Chimeric antigen receptor (CAR) T cells are the first example of a “living drug”. 1 Following in vitro production from T cells collected from a patient’s leukapheresis procedure, the CAR T cells are infused back into the patient’s blood, where they proliferate and expand. This is variable and to some extent unpredictable.
Pharmaceutical Technology
SEPTEMBER 30, 2022
After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. 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.
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Pharmaceutical Technology
APRIL 13, 2023
Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.
pharmaphorum
JUNE 12, 2022
bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8
pharmaphorum
APRIL 14, 2022
US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. gene therapy is cost-effective appeared first on. The post ICER says bluebird bio’s $2.1m
Pharmaceutical Technology
APRIL 5, 2023
More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.
pharmaphorum
FEBRUARY 16, 2021
It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells. million in Europe.
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