Vertex Pharma

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A giemsa stained blood smear from a person with beta thalassaemia

First CRISPR drug Casgevy gets swift second approval

The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-

News

CHMP backs Biogen’s Skyclarys for Friedreich’s ataxia

Biogen’s Skyclarys is on course to becoming the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU after it was recommended

News

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

News

FDA approves first cell therapy for type 1 diabetes

CellTrans has claimed the first FDA approval for a cell therapy used to treat type 1 diabetes, consisting of insulin-producing islet beta cells taken from deceased donors.

News

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The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

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