Dear Duchenne Community,
As we begin the new year, we want to wish you and your families a happy and healthy 2026 and to thank you for your continued partnership. We are writing to share an update on SGT-003, our investigational next-generation gene therapy for Duchenne muscular dystrophy.
Today, we announced that 33 participants have been dosed (ages 1-10, as of January 9, 2026) in INSPIRE DUCHENNE, our Phase 1/2, open-label, multi-center clinical study evaluating the safety, tolerability, and efficacy of SGT-003.
We are proud to stand in partnership with the Duchenne community and remain committed to sharing timely, transparent safety information for every participant who receives SGT-003 so families and clinicians can make informed decisions. That openness and accountability are core to how we conduct this program. As of the January 9, 2026, cutoff, SGT-003 has been generally well tolerated using prophylactic steroids alone for immune suppression. No cases of drug-induced liver injury (DILI), thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS) or myocarditis have been observed. Cardiac monitoring has shown consistent reductions in markers of cardiac injury with early signals of normalization, and we have observed compelling microdystrophin expression and improvements across multiple biomarkers of muscle health. Additional information may be found in a company press release issued today.
The unmet need in Duchenne remains high, and we feel a deep responsibility in advancing SGT-003. To support the path forward, we are pursuing an integrated U.S. and ex-U.S. pivotal trial strategy to support potential accelerated regulatory authorizations. We are pleased to share that we have enrolled the first patient in IMPACT DUCHENNE, our Phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy of SGT-003, and expect to initiate dosing in the first quarter of this year. As previously announced, IMPACT DUCHENNE will be conducted outside the United States. The first clinical trial sites have been activated in Canada and Australia, and we plan to expand to European countries beginning mid-year 2026, subject to receipt of regulatory approvals.
We would like to express our sincere gratitude to the patients and families who choose to participate in clinical research. We recognize the time, energy, trust, and courage these decisions require, and we are deeply thankful for the partnership and commitment families bring to advancing the field for the entire Duchenne community.
We remain committed to sharing updates clearly and in a timely manner. The most up-to-date information for both clinical studies may be found on ClinicalTrials.gov.
- INSPIRE DUCHENNE (NCT06138639)
- IMPACT DUCHENNE (NCT07160634)
An additional resource for our ongoing research may be found here: https://www.solidbio.com/patient-caregiver-resources/resources-policies/clinical-trials
We are looking forward to connecting with the Duchenne community in person in the coming months, including at:
- Duchenne UK’s New Horizons Conference, February 6–7 in London, U.K.
- Parent Project’s 23rd Annual International Duchenne and Becker Conference, February 27–March 1 in Rome, Italy
If you have questions or would like to be in touch, please reach out to our patient advocacy team at community@solidbio.com.
Thank you again for your trust and partnership.
With gratitude,
Annie Ganot
Co-founder, SVP Patient Advocacy
Solid Biosciences