beta thalassaemia

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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-

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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

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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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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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engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Vertex, CRISPR prep filing for gene-editing blood disorder t...

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this

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bluebird unveils $2.8m price for gene therapy Zynteglo on FD...

bluebird bio's Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require re