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FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

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bluebird bio presents positive results for inherited blood disorder gene therapies

The company’s sickle cell disease therapy was accepted for FDA priority review earlier this year

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bluebird bio to sell FDA priority review voucher for $103m

The company previously sold its second voucher to Bristol Myers Squibb for $95m

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bluebird bio’s sickle cell gene therapy accepted for FDA priority review

The genetic disease affects approximately 100,000 people in the US

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bluebird bio submits US application for sickle cell gene therapy

The genetic disease affects approximately 100,000 people in the US alone

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bluebird bio sells second FDA priority review voucher for $95m

The company sold its first voucher to Dutch biotech Argenx for $102m

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FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

The company can now resume the enrolment and treatment of patients aged two to 17 years

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bluebird bio sells Rare Pediatric Disease Priority Review Voucher

The company was granted two PRVs upon the FDA approvals of Zynteglo and Skysona

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bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

The gene therapy was shown to slow the progression of neurologic dysfunction

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bluebird bio’s beta-thalassaemia gene therapy receives FDA approval

Patients with the most severe form of the blood disorder develop life-threatening anaemia and have to receive regular blood transfusions

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bluebird bio reveals plans to launch two gene therapies

Both therapies were endorsed by the US FDA Cell Tissue & Gene Therapy Advisory Committee

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bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company plans to reduce its workforce amid efforts to reduce its operating costs

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