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tag myelodysplastic-syndrome

Zynteglo halt re-ignites viral vector safety concerns; analysts

pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS).

Gene Therapy

Gene Therapy Gene Gene Sequencing Gene Expression

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

XTalks

SEPTEMBER 27, 2022

The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. The price tag reflects “the clinical benefit [it] provides as an urgently needed treatment option to slow the progression of neurologic dysfunction in children impacted by progressive, irreversible and fatal rare disease,” Obenshain added.

FDA Approval

FDA Approval Gene Therapy Drugs Gene

Zynteglo halt re-ignites viral vector safety concerns; analysts

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

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