sickle cell disease

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Vertex Pharmaceutical's Boston headquarters

FDA clears two sickle-cell gene therapies, including CRISPR ...

The US FDA has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.

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UK first to approve Vertex, CRISPR drug for blood disorders

The first-ever CRISPR-based gene-editing therapy has been approved for marketing in the UK for inherited blood disorders sickle cell disease (SCD) and transfusion-dependen

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FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

The FDA has started a priority review of Vertex Pharma and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD), with a decision on approva

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EU approval for Novartis’ sickle cell drug set to be pulled

Novartis has said it is “disappointed” that the EMA’s Human Medicines Committee has recommended revoking the conditional EU approval for sickle cell disease (SCD) therapy

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ICER backs sickle cell gene therapies at one-off cost of $2m

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

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