Solid Biosciences Announces 2026 Outlook for Clinical Trials of Duchenne Gene Therapy SGT-003

In a press release issued by Solid Biosciences, it was stated that “With multiple regulatory interactions planned in the coming months, we aim to achieve alignment on a potential accelerated approval pathway for SGT-003 and to bring our investigational therapy to market to meet the overwhelming demand from the Duchenne community.”” Learn More: Frequently Asked Questions About Solid Biosciences SGT-003 Gene Therapy

SGT-003 for Duchenne muscular dystrophy (Duchenne) in USA

• As of a safety cutoff date of January 9, 2026, SGT-003 has been generally well tolerated in the 33 participants dosed in the ongoing INSPIRE DUCHENNE Phase 1/2 clinical trial.
  • SGT-003’s safety and tolerability profile continues to support a steroid-only prophylactic immunomodulation regimen.
  • Outpatient dosing has been enabled since September 2025.
  • No drug-induced liver injury (DILI), thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS) or myocarditis observed as of the January 9, 2026, cutoff date.
  • Cardiac safety monitoring has shown reductions in cardiac injury and early signals of cardiac systolic function normalization, as measured by left ventricular ejection fraction (LVEF) (N=14, data cutoff of September 29, 2025).
• Compelling microdystrophin expression levels (N=10, data cutoff of September 29, 2025) and concordant restoration of key components of the dystrophin-associated protein complex (DAPC) have been observed.
• Improvements across a range of biomarkers of muscle integrity have been observed suggesting a coordinated downstream effect (N=11-14, data cutoff of September 29, 2025).
• During the first half of 2026, the Company plans to have multiple interactions with the U.S. Food and Drug Administration (FDA) to align on its Phase 3 confirmatory trial design and on necessary confirmatory evidence required to support potential accelerated approval for SGT-003, with an update expected mid-year 2026.
• The Company expects to report additional data from the Phase 1/2 INSPIRE DUCHENNE trial in mid-2026.

Discover More: Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males

Clinical Trial of SGT-003 outside the USA

• The first participant has been enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected to occur in the first quarter of 2026.
• The IMPACT DUCHENNE trial has two active clinical trial sites, located in Canada and Australia, with planned expansion into additional countries, including in Europe, beginning in mid-year 2026, subject to the receipt of regulatory approvals.
• SGT-003 has been awarded the Innovation Passport Designation under the new U.K. Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market and facilitate patient access to new medicines, positioning SGT-003 to potentially become the first-to-market Duchenne gene therapy in the U.K. >>> Innovation Passport Designation

Read More: Clinical Trials for Duchenne (List of All Researches)

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