DMD research

Solid Biosciences report positive data from ongoing DMD gene therapy trial

Life sciences company Solid Biosciences have released data from Phase I/II of their ongoing clinical trial of their gene therapy for Duchenne muscular dystrophy (DMD) which shows encouraging evidence that the treatment is benefitting patients 12-24 months after their first dose.  

Gene therapy for DMD involves delivering new genetic material to cells to overcome errors (or mutations) on the dystrophin gene. It is one of the most promising treatments on the horizon and huge progress has been made in the last few years, with clinical trials now taking place in the UK and abroad. In 2014, Duchenne UK partnered with the Duchenne Research Fund and Joining Jack to commit $5 million to help fund preclinical work for Solid’s gene therapy programme. 

Read more about gene therapy and the research that Duchenne UK is funding in this area here. 

The data from Solid Bioscience’s trial, called IGNITE-DMD, reported continued functional benefit 1.5 years post-treatment, with patient reported outcomes also showing sustained improvement. This data was gathered by comparing trial participants’ functional motor abilities against natural history data (the natural progression of the disease without treatment) using the North Star Ambulatory Assessment. It showed that the patients’ motor abilities declined less than what is typically expected in DMD. They were also able to maintain their 6-minute-walk distances over the trial period, whereas similarly-aged patients’ abilities would typically decline over this time. There were also no new safety findings from patients 3.5 years post-dosing.  

Read the full press release from Solid Biosciences here. 

  

Want to understand the terminology used when we talk about DMD research? Visit our research glossary. 

Published on 8 October 2021

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