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tag inherited-disease

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

FDA

FDA Insurance Patients Marketing

Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

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). The standard of care for the condition includes blood transfusions, hydroxyurea and iron chelation.

Healthcare

Healthcare Healthcare Pharmaceutical FDA

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

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