Solid Biosciences has shared an update on SGT-003, its investigational gene therapy for Duchenne muscular dystrophy (DMD), coinciding with presentations at the annual Parent Project Muscular Dystrophy (PPMD) conference in the United States.
What is SGT-003?
SGT-003 is an investigational gene therapy being developed by Solid Biosciences. It uses an adeno-associated virus (AAV) to deliver an engineered version of the dystrophin gene directly into muscle cells. The aim is to enable the body to produce a shorter, but functional, form of the dystrophin protein that is absent or severely reduced in Duchenne.
INSPIRE DUCHENNE: Enrolment and Safety Update
Solid Biosciences has confirmed that 53 boys have now been dosed with SGT-003 in the INSPIRE DUCHENNE Phase 1/2 clinical trial, as of a June 2026 enrolment cutoff. Participants span a broad paediatric age range from 0 to 10 years old, including one child aged just six months. This is reported to be the youngest child dosed to date with a microdystrophin gene therapy.
On safety, the update reports that SGT-003 has been generally well tolerated across participants. There have been no observed cases of drug-induced liver injury (DILI), myocarditis, thrombotic microangiopathy (TMA) or atypical hemolytic uremic syndrome (aHUS). All participants received a steroid-only prophylactic immunomodulation regimen alongside SGT-003, a regimen designed to support tolerability while minimising treatment burden on patients and families.
IMPACT DUCHENNE: Phase 3 Trial Underway
Solid Biosciences has confirmed that IMPACT DUCHENNE, its Phase 3 randomised, placebo-controlled trial evaluating SGT-003, is now underway. The trial is currently enrolling participants at clinical sites in Australia and Canada, with clinical site expansion planned in the United States.
What This Means for UK Families:
For families in the UK, the most significant development in this update is the planned expansion of the Phase 3 IMPACT DUCHENNE trial to UK clinical sites. While timings remain subject to regulatory authorisation, this represents a meaningful step towards UK families being able to access this trial.
We will continue to monitor developments and share updates as they become available.
Questions or Concerns?
If any of the information in this article has raised questions or concerns, please do not hesitate to get in touch. Action Duchenne is here to support you and your family every in their journey.
Email us: Support@actionduchenne.org
