In this issue of Deep Dive we explore the ever present issues of market access and commercialisation, analysing recent changes in China, England and the US and asking how pharma can work with he
A European Medicines Agency advisory panel has recommended a conditional approval for Zynteglo, bluebird bio’s lead gene therapy for thalassemia, a rare blood disorder.
The scientific advancements of the past 30 years have transformed our understanding of the potential of gene therapy and how it may be used to tackle rare diseases.
Patients with the rare blood disorders transfusion-dependent β-thalassemia (TDT) have shown spectacular results in clinical trials of bluebird bio's gene therapy, raising hopes the treatmen