Tom Oakley
Tom Oakley is Principal Consultant at Somerville Development Partners.
JournalThe first approval of a CRISPR-Cas9 gene editing medicinal product in the European Union: Casgevy
Patients with transfusion-dependent β-thalassemia and severe sickle cell disease suffer the burden of repeated blood transfusions and vaso-occlusive crises, respectively. Casgevy (exagamglogene autotemcel) consists of autologous CD34+ haematopoietic stem and progenitor cells edited, ex vivo, by CRISPR-Cas9 technology. Gene editing using Casgevy results in reduced expression of the BCL11A gene, ...