Gene therapy trial markedly cuts bleeding risk in haemophilia B patients

The risk of bleeding in people with haemophilia B has been slashed by a new gene therapy.

The need for people to inject themselves weekly with clotting factors was eliminated by a single injection of the gene therapy. The study was led by experts from the University College London.

The ability to clot is affected by hemophilia. It is usually passed on to men. When a person cuts themselves, clotting factors mix with blood cells to stop the bleeding. People with haemophilia are at risk of heavy bleeding.

A lack of the blood-clotting factor VIII is the main cause of haemophilia A, while a deficiency of factor IX is the main cause of haemophilia B.

To make up for the deficiency in clotting factor IX, people with haemophilia B need to inject themselves weekly.

Nine out of 10 patients with severe or moderately severe haemophilia who received a single treatment with FLT180a had sustained production of the protein from the liver, according to the New England Journal of Medicine.

The need for regular injections was eliminated by this.

The study found that removing the need for haemophilia patients to inject themselves with the missingprotein is an important step in improving their quality of life. The long-term follow-up study will keep an eye on the patients for the long term.

The patients had to take immune-suppressing drugs for several weeks to prevent their immune systems from rejecting the therapy.

One patient who received the highest FLT180a dose had an abnormal blood clot, which was caused by the high levels of theprotein.

The co-author of the study said thatGene therapy is still a new field that pushes the boundaries of science for people with severe genetic diseases.

The trial added to the growing body of evidence that gene therapy has the potential to free patients from the challenges of having to adhere to lifelong therapy.