logo
Menu

This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Drugs > CTX001 for transfusion-dependent beta (β)-thalassaemia

< Back

CTX001 for transfusion-dependent beta (β)-thalassaemia

Drugs

Haematology and Blood Products

September 2021


CTX001 is under clinical development for the treatment of patients with beta-thalassaemia who require blood transfusions. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body. Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
CTX001 is an ex vivo CRISPR investigational gene-edited therapy that is administered intraveneously. The patient’s haematopoietic stem cells are genetically engineered to produce high levels of foetal haemoglobin (HbF; haemoglobin F) in red blood cells. The elevation of HbF by CTX001 has the potential to improve transfusion requirements for beta-thalassaemia patients. If licensed CTX001 will offer a treatment for patients with transfusion-dependent beta-thalassaemia.

Innovation Observatory Voice 0

Leave a Reply

Your email address will not be published. Required fields are marked *

Post Comment

Download Full Article



 

Connect to the Innovation Observatory

Twitter

Load More Related Posts

Get Alerts