Recommendations for gene therapy trial preparedness published in Journal for Neuromuscular Disorders

The recommendations are the result of a meeting hosted by Duchenne UK and the DMD Hub to discuss the challenges and opportunities of gene therapy. The UK has some of the world’s leading clinicians and a wealth of research in both clinical and academic institutions, making it an attractive location for gene therapy trials. However, several challenges exist if trials are to be set up and run here.

More than 100 stakeholders, including patient groups, clinicians, and representatives from industry attended the meeting, alongside NICE, NHSE and government funded initiatives that are working to facilitate gene therapy.

Emma Heslop, DMD Hub Manager said 'The DMD Hub meeting was very timely and the agreed actions have enabled the DMD Hub to establish areas of work which will facilitate the efficient set up and effective delivery of DMD gene therapy trials in the UK in 2021'. 

Some of the challenges that the DMD Hub is now working to address include understanding patients’ views around taking part in a gene therapy trial, establishing a fair and equitable trial recruitment process and assessing institutional readiness of the hospitals potentially able to run the gene therapy trials. 

Alex Johnson from Duchenne UK said: “Families in the UK are eager to take part in gene therapy trials. Despite the challenges of Covid-19, gene therapy trials are moving ahead and we are keen to maintain the momentum of setting up trials in the UK.”

The discussion and findings from the meeting have been published in the Neuromuscular Disorders Journal and are available to view on the DMD Hub Toolkit.

The DMD Hub will continue to work collaboratively with patients and their families, sites and industry to help ensure that DMD gene therapy trials in the UK become a reality in 2021.