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
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
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
UK biotech Silence Therapeutics has launched an online game to raise the profile of the rare blood disorder thalassaemia, and one of its most common symptoms.
bluebird bio is pricing its thalassaemia gene therapy Zynteglo at €1.57 million ($1.8 million) in Europe, but with an outcomes-based payment scheme to try to persuade healthcare systems to