A groundbreaking gene therapy treatment for the bleeding disorder haemophilia B has been introduced to the NHS, offering hope to patients like Elliott Collins who have struggled with the condition for years.

Elliott Collins, a 34-year-old from Colchester, spent nearly three decades relying on regular injections of clotting factor IX to manage his haemophilia. Without enough of this crucial clotting component, bleeds were more severe and could occur spontaneously in joints, causing long-term damage.

However, Elliott was among the first participants in trials for the gene therapy treatment that has now proven successful. This innovative therapy, with an official cost of £2.6 million, has completely transformed Elliott’s life. He no longer requires factor IX injections and has experienced a significant improvement in his condition.

Describing his experience, Elliott shared, ‘I don’t think you can go through something like that for 29 years, for it to completely disappear and not change someone mentally and physically. I feel cured.’ His realization that the treatment was effective came when he injured his knee but only suffered a minor mark instead of a severe bleed.

The gene therapy treatment involves using engineered viruses to deliver correct instructions for producing factor IX to the liver. By correcting the genetic mutation that caused the deficiency, the therapy has successfully increased factor IX levels in Elliott’s blood to 60% of normal.

This breakthrough in gene therapy offers new hope to individuals with haemophilia B, providing a potential alternative to regular injections and improving their quality of life. The successful results seen in patients like Elliott Collins highlight the promising future of gene therapy in treating genetic disorders.