Key milestone: first UK patient enrolled in DMD gene therapy trial at DMD Hub site

Pfizer has announced that the first UK-based DMD patient has been enrolled in a gene therapy trial at Newcastle Hospitals, a DMD Hub site.

Pfizer have announced the enrolment of their first participant in a Duchenne muscular dystrophy (DMD) gene therapy trial in the UK. This is the first time a DMD patient in the UK has received gene therapy, and is a landmark moment in the mission to treat the condition. The patient has been enrolled at Newcastle Hospitals; a DMD Hub site, and hosts to the NIHR Newcastle BRC.

The NIHR Newcastle Biomedical Research Centre’s Neuromuscular Disease theme supports the work of researchers dedicated to finding treatments for people living with DMD. Deputy Lead for the theme and Professor of Neuromuscular Genetics at Newcastle University, Volker Straub comments:

It is outstanding how far work in this area has come. Only 20 years ago it was hard to believe that we could effectively treat a degenerative genetic disease, but we are now involved in work like this, with hopefully some amazing outcomes.

The patient was enrolled at Newcastle, one of three locations in the UK where the Gene Therapy study is taking place. For more details on the trial in the UK, please see here

The trial is a Phase 3 global, multicentre, randomised, double-blind, placebo-controlled study. In total, 99 boys across 55 sites in 15 countries will take part in the trial, with the first patient dosed in Barcelona in December 2020. It will evaluate the efficacy and safety of gene therapy construct PF-06939926, with participants being ambulatory boys between the ages of 4 and 7.

Dr Michela Guglieri, Consultant Neurologist at Newcastle Hospitals and UK Chief Investigator of the study, said:

The enrolment of the first UK patient in this Phase 3 gene therapy programme is a great achievement for the UK Duchenne community and we are very pleased to contribute to innovative research in the pursuit of future therapies. There are currently no approved disease-modifying treatment options available for all genetic forms of the disease in the UK. I’m very proud to be leading the UK arm of this global study.

You can read the full press release from Pfizer here