Dear Duchenne Community,
We are pleased to share a meaningful update on the INSPIRE DUCHENNE clinical study, our multi-center, open-label clinical trial evaluating SGT-003, our investigational gene therapy for the treatment of Duchenne muscular dystrophy.
As of August 12, 2025, 15 patients living with Duchenne have received SGT-003 in this study. The trial now includes 10 active clinical sites in the United States, Canada, Italy, and the United Kingdom, with additional sites expected to open later this year. Based on the strong interest we continue to receive from the Duchenne and clinical communities, we expect to have treated a minimum of 20 patients, aged 0 to less than 12, by the end of 2025. We anticipate and look forward to expanding enrollment into older aged patients in 2026.
To date, SGT-003 has been well tolerated. No patients have experienced serious adverse events related to treatment. There has been one case of a mild increase in liver enzymes after dosing, which occurred during the steroid taper period. This participant has shown no symptoms, has experienced no changes to daily activities, and is doing well on steroids alone.
We recognize that the urgent need for safe and effective therapies for Duchenne persists. That urgency drives us to advance SGT-003 as quickly and safely as possible, with patients and families at the heart of every decision. We are grateful to those participating in this research for making progress possible.
Later this year, in the fourth quarter of 2025, we anticipate meeting with the U.S. Food and Drug Administration (FDA) to discuss regulatory pathways for SGT-003. Additionally, we intend to initiate a separate randomized, placebo-controlled trial evaluating SGT-003 outside of the United States, also expected in the fourth quarter of 2025, with the aim of supporting potential global regulatory authorizations as we strive to bring Solid’s next-generation gene therapy to those who need it, globally.
For more information on the study, including participating sites, please visit clinicaltrials.gov – INSPIRE DUCHENNE – NCT06138639.
We will continue to share updates as soon as they become publicly available. If you have any questions, please reach out to us at community@solidbio.com.
With gratitude,
Annie Ganot
Co-founder, SVP Patient Advocacy
Solid Biosciences
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