Defeat Duchenne Canada is pleased to share with you some updates from Solid Biosciences, currently running two gene therapy clinical trials in Duchenne muscular dystrophy (DMD). Full community letter and recent press release can be found below.  

• INSPIRE TRIAL: So far, this multicentered phase 1/ 2 trial investigating a single dose of SGT-003 in ambulant boys with DMD has enrolled 33 children between the ages of 1-10 years of age. The gene therapy treatment has generally been well tolerated using steroids alone for immune suppression and no serious safety concerns identified thus far. Results show an increase in micro-dystrophin expression. Micro-dystrophin is a scaled-down version of the large gene that encodes dystrophin. There have also been improvements in multiple markers of muscle and heart health noted as well. SGT-003 is an adeno-associated virus (AAV) based gene therapy. 

• IMPACT TRIAL – So far, this randomized, double-blind, placebo-controlled study (where participants are assigned to receive SGT-003 or placebo, and nether participants or their investigators know who is receiving the drug) has enrolled its first participant into this phase 3 trial. There are clinical trial sites active in Canada (in British Columbia) and Australia with plans to expand to Europe later this year. 

The company remains committed to sharing results and progress with the DMD community. And we look forward to sharing more updates with you as they become available. 

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