With the funds we raised at our 2013 Gala Dinner we brokered the first partnership between UK Duchenne charities and commercial enterprise to launch Solid GT, an exciting gene therapy project. We approached US-based Solid Biosciences to present promising gene therapy research we had funded, and to accelerate the option of gene therapy as a treatment for Duchenne.

The DRF then collaborated with the Duchenne Children’s Trust and Joining Jack to contribute $5 million to launch Solid GT. Early stage pre-clinical research has already had encouraging results, putting the project on track to enter the clinical trial phase within two years of its launch.

Gene therapy is one of the most exciting prospects for stopping Duchenne muscular dystrophy in its tracks. Drugs we have previously funded show promise in slowing muscle deterioration. But a successful gene therapy treatment would deliver a therapeutic gene into cells to replace the faulty gene. It could help beat Duchenne for all boys, regardless of type of mutation. Click here to read more about all our projects.

Solid Biosciences CEO Ilan Ganot said: ‘Solid GT is excited to have partnered with three UK charities, who are playing key roles in enabling our pre-clinical development efforts as well as facilitating dialogues with stakeholders.’