Solid Biosciences has issued a press release about their gene therapy clinical trial. They treated the first boy with Duchenne about one month ago and a few days after treatment they found some abnormalities in his blood test results, which have now improved. These kinds of events need to be reported to the regulatory authorities (the FDA) and the trial has been temporarily halted for further investigations.
Gene therapy involves delivering a functional version of the dystrophin gene to the muscles using a harmless virus called AAV (adeno-associated virus). This research is particularly attractive because it has the potential to benefit the majority of boys (and rare girls) with Duchenne no matter what their genetic mutation is.
We have spoken to the team at Solid and they have reassured us that the boy treated is well and is back at home with his family.
We hope that this is only a small hiccup and recruitment for the trial will continue soon. Solid are happy to answer any questions from the Duchenne Community, so please email your questions to firstname.lastname@example.org and we will compile and send to them.
You can read the press release here:
Solid has also issued a letter to the community: