What We Do Funding Research & Trials Solid Biosciences Gene Therapy Programme Our Partnership In 2014, Duchenne UK and the Duchenne Research Fund invested $5million in Solid GT (a subsidiary of Solid Biosciences) to help them develop a gene therapy programme. As a result of that investment, Solid GT was able to generate data that led to a financing round last year, where they raised $42million. We as charities could not have raised that. But our investment - early in the process and a relatively high risk for other big investors - helped to drive the company to raising the money they needed. And we continue to work with Solid to give advice as patients and as advocates. We are on this journey with them. Andrey Zarur, Founder and Chairman of Solid Biosciences, said: “For the first time in history, Duchenne muscular dystrophy will have a fully-funded platform to explore gene therapy through a rigorous scientific and clinical process.” What Is Solid GT Working On? The company’s licensed technology has demonstrated benefits in various DMD animal models, and lead candidates are currently undergoing Investigational New Drug studies in preparation for clinical trials. Solid GT collaborates with leading patient organisations, experts, universities, hospitals and gene therapy centres, globally. One of these is Prof Dongsheng Duan who has used a safe virus known as Adeno Associated Virus (AAV) to deliver a mini version of the missing dystrophin gene to a dog with the disease. Please visit www.solidbio.com for more information. Read more on gene therapy here.