Haematopoietic stem cell therapies: gene addition, editing and molecular analysis    

Over the last 20 years, increasing numbers of patients have shown remarkable clinical effects following gene therapy. Some of these therapies are being developed as licensed medicines and we are developing new treatments for rare diseases where existing therapies are either dangerous or not completely effective. 

We want to use gene therapy for patients with inherited problems of the blood and immune system. We have pioneered some of this work and we aim to improve the technology to enhance safety and ensure that effectiveness is reliably achieved in all patients. We want to make sure that the treatments can cover a broader number of diseases and be extended to more patients. 

Our hope is that haematopoietic stem cell therapies will become part of the standard care pathway for patients with devastating diseases.