sickle cell disease

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The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

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Vertex/CRISPR file first gene-editing therapy with FDA

Vertex Pharma and CRISPR Therapeutics have become the first companies to file for FDA approval of a therapy based on gene-editing technology.

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EMA reviewing Novartis' Adakveo after phase 3 miss

The EMA's human medicines committee has started a review of Novartis' sickle cell disease (SCD) therapy Adakveo, shortly after the company reported that it failed to show efficacy in a phas

UK first to approve Vertex, CRISPR drug for blood disorders

FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

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

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

Vertex/CRISPR file first gene-editing therapy with FDA

EMA reviewing Novartis' Adakveo after phase 3 miss

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sickle cell disease

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