Rethinking Clinical Trials

FEBRUARY 20, 2024

Chair in General Internal Medicine Rutgers, Robert Wood Johnson Medical School Keywords Transfusion; MI; MINT; Anemia; Clinical trials Key Points Anemia is common in patients with acute MI. Due to the lack of evidence, indications for red blood cell transfusion in patients with MI are controversial.

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Trials Clinical Trials Clinical Trials Medicine 130

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.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

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. million if paid through an outcomes-based contract for patients with sustained transfusion independence.”

Gene Therapy 62

Gene Therapy Gene Marketing Clinical Trials 62

XTalks

DECEMBER 13, 2023

Sickle cell disease is a group of inherited red blood cell disorders that affect approximately 100,000 people in the US, most of whom are Black. The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

pharmaphorum

JUNE 12, 2022

The panel voted 13 to 0 on the question of whether beti-cel’s benefits outweighed the risks of the gene therapy in patients with beta thalassaemia who are dependent on blood transfusions, in a dream result for the biotech which just a few weeks ago was expressing doubts about its ability to continue as an operating concern.

Gene Therapy 57

Gene Therapy Gene Marketing Trials 57

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.

Gene Therapy 147

Gene Therapy Gene FDA Approval Licensing 147

XTalks

AUGUST 31, 2022

Ayesha shared news about another significant FDA approval for a gene therapy to treat transfusion-dependent beta thalassemia. Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder.

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Gene Therapy FDA Approval Life Science Gene 52