Solid Biosciences has announced that the next stage of their clinical trial of a gene therapy has started, following the dose increase announced in February.
The early stage phase 1 clinical trial is testing the safety of a potential gene therapy called SGT-001. During the trial, each participant receives a single dose of the potential therapy. Through the trial, the dose is gradually increased, and this second part of the trial is using doses 4 time higher than the first part. This comes after the trial safety monitors gave the go-ahead in February.
So far, 2 people have been recruited to this part of the trial and one has received SGT-001. The individual had similar short-lived side effects to previous participants but tests of liver function showed some abnormalities. These were treated with steroids and the participant recovered fully and is doing well.
The company has also announced plans to open a second trial site. They hope that the site at the University of Massachusetts in the US will start recruiting shortly.
We believe that SGT-001 has the potential to be an important therapy for patients with Duchenne, and we are working hard to progress SGT-001 through clinical development quickly and responsibly. We look forward to continuing to keep you updated as the study advances.
Solid Biosciences Team
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