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The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from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