First patient treated in global gene therapy trial at Manchester CRF at MRI

News posted 21 August, 2019

The NIHR Manchester Clinical Research Facility (CRF) at Manchester Royal Infirmary has successfully dosed the first patient in the UK and the first patient to Cohort 2 globally to its phase 1 gene therapy trial to help improve the lives of patients with severe haemophilia A.

The GET8 research trial is being led locally by Chief Investigator Charles Hay and the Non-Malignant Haematology Research Team at NIHR Manchester Clinical Research Facility (CRF) at Manchester Royal Infirmary. The team have recruited and dosed the first patient to GET8 which is being trialled in human volunteers for the first time across 25 research sites globally.non_malignant-research-team

Haemophilia A is a hereditary bleeding disorder whereby a protein that helps the body to make blood clots is either partly or completely missing. Although it is the most common type of haemophilia it is a rare condition mainly affecting around one in 10,000 males. People with severe haemophilia may bleed one to two times per week into their muscles or joints causing pain, swelling and limited movement and have a tendency to bruise easily and bleed excessively from cuts.

Current treatment for people living with this condition replaces the missing clotting factor VIII in the blood and requires frequent injections on a daily basis for life. GET8 aims to reduce the amount of daily injections by determining the safety and tolerability of a factor VIII gene transfer treatment (a single infusion of adeno-associated virus (AAV) containing DNA with relevant parts of the factor VIII gene).

This trial is supported by the NIHR Greater Manchester Clinical Research Network and funded by Bayer PLC.