The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from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 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
Blood cells grown in a laboratory have been given to people for the first time in a clinical trial being carried out by researchers in the UK, in the hope that plentiful supplies of rare bl
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
Novo Nordisk has doubled down on position in the sickle cell disease (SCD) category with a $1.1 billion agreement to acquire Forma Therapeutics, and its therapeutic candidate etavopivat, in