United States – October 27, 2026 -- Solid Biosciences has expanded its INSPIRE DUCHENNE gene therapy trial to 43 participants and now accepts children aged 4 to under 12, up from the previous range of 4 to under 8.
Three participants dosed with no serious adverse events
INSPIRE DUCHENNE is a first-in-human, multicenter, Phase 1/2 open-label study. It tests the safety, tolerability and efficacy of a single intravenous infusion of SGT-003, a next-generation AAV gene therapy candidate, in ambulant boys with Duchenne.
Three participants have been dosed so far. According to Solid, SGT-003 has been well tolerated to date, and no serious adverse events have been reported. The company plans to share safety, expression and biomarker data from these first three patients, along with a trial update, in the first quarter of 2025.
Functional endpoints now measured at 18 months
Citing encouraging early results, Solid also moved the evaluation of functional endpoints from 12 months to 18 months. The company says the longer window should better detect whether SGT-003 can change the course of the disease.
Three sites enrolling now, three more coming
Solid has activated one additional site and plans to add three new clinical sites across the U.S. and Canada before the end of the year. Actively enrolling sites are:
- Nationwide Children's Hospital, Columbus, OH
- University of California Los Angeles Medical Center, Los Angeles, CA
- University of California Davis Medical Center, Sacramento, CA
Upcoming sites are:
- Arkansas Children's Hospital, Little Rock, AR
- Children's Hospital of the King's Daughters, Norfolk, VA
- The Hospital for Sick Children (SickKids), Toronto, Canada
The company also plans to extend the study into Europe during the first half of 2025, with the aim of giving more families the chance to participate.
Three community webinars scheduled in November
Solid is working with three advocacy organizations on webinars where its Clinical and Patient Advocacy teams will explain more about the trial. All start at 1 p.m. EST:
- Parent Project Muscular Dystrophy: Tuesday, November 12, 2024
- Defeat Duchenne Canada: Wednesday, November 13, 2024
- Jett Foundation: Thursday, November 21, 2024