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
Anyone who has taken a psychiatric medication knows that the status quo in prescribing is a trial and error approach, with patients often cycling through an array of drugs to find the one t
It’s 2024, and in most clinical trials, data is still being moved from the EHR to the EDC manually – with employees reading data from one screen and typing it onto another.