Solid Biosciences has been given the green light to continue their Duchenne gene therapy trial.
The FDA originally placed a hold on the trial in March this year because the first patient dosed with the gene therapy experienced a serious adverse event which was picked up in blood tests. This was identified early and well-managed and has now fully resolved. The young man has returned to his normal activities.
Solid plans to resume recruitment as soon as possible with some changes to the trial protocol. This includes a modified steroid regimen and additional monitoring measures, as well as guides to facilitate early medical interventions should an event occur. They have also stated that although the trial is for 4 to 17 year olds, they plan to treat several younger patients next before resuming with non-ambulant adolescents.
This is great news, and we look forward to hearing the first results from the trial.
Save Our Sons Duchenne Foundation has granted $900K to Solid Biosciences over the past few years to help support research into gene therapy and other promising approaches for Duchenne.
- More information about Duchenne [link to https://www.saveoursons.org.au/what-is-dmd/]
- Read the Solid Biosciences press release https://www.solidbio.com/content/solid-biosciences-announces-fda-removes-clinical-hold-sgt-001
- Solid’s letter to the community https://www.solidbio.com/content/clear-resume-ignite-dmd
- More information about the trial https://clinicaltrials.gov/ct2/show/NCT03368742?term=Solid+Biosciences&cond=Duchenne+Muscular+Dystrophy&rank=1