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-
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
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
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
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
The FDA has approved Akebia Therapeutics’ vadadustat as a treatment for anaemia caused by chronic kidney disease (CKD) in dialysis patients, becoming the first rival to GS