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 > Luspatercept for the treatment of adults with non-transfusion dependent beta (β)-thalassaemia

< Back

Luspatercept for the treatment of adults with non-transfusion dependent beta (β)-thalassaemia


Haematology and Blood Products

August 2020

Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t regularly require blood transfusion. 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.
Luspatercept, administered via subcutaneous injection, is a recombinant engineered protein designed to attach to certain proteins that slow down the maturation of red blood cells. This leads to the production of healthy red blood cells and increased haemoglobin levels, leading to improved symptoms in patients with beta-thalassaemia intermedia and major. Luspatercept is a novel approach for treating anaemia, with potential to improve many patients’ lives by reducing or eliminating the need for frequent and lifelong blood transfusions.

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Your email address will not be published.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


Load More Related Posts

Get Alerts