We were told gene therapy would not happen in our lifetimes. But we did not accept that. In 2014, Duchenne UK partnered with the Duchenne Research Fund and Joining Jack to commit $5million to help fund pre-clinical work for Solid Biosciences’ 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.

Just three years later, their compound is IN THE CLINIC being tested on boys with DMD! We are extremely proud to have played a part in helping to accelerate the development of this gene therapy treatment. We await with hope the results of the study.

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 Biosciences 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.