In a press release yesterday, Solid Biosciences gave a short update on their gene therapy trial called Ignite DMD. Following the decision earlier this year to move to the higher dose of the potential treatment, a second participant has received the higher dose of SGT-001.
The trial is an early stage trial, which is testing the safety of SGT-001 in people living Duchenne. SGT-001 is a potential gene therapy that uses a harmless virus to deliver a smaller version of the dystrophin gene (called a microdystrophin) to cells.
Solid also announced that they have made some changes to the trial plan. The company hopes these changes, which include adding a weight limit for trial participants and removing the control arm from the trial will help to ensure that the results of the Ignite DMD trial are available as quickly as possible.
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