SGT-003 / Solid BioSci - LARVOL DELTA

SGT-003 gene therapy restores DAPC stability and improves muscle integrity in Duchenne muscular dystrophy (ESGCT 2026) - No abstract available

Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Phase I/IIa Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, Immunogenicity, and Preliminary Efficacy of SGT003 in Patients With Advanced Solid Tumors (clinicaltrials.gov) - P1/2 | N=332 | Recruiting | Sponsor: Beijing Sungen Biomedical Technology Co., Ltd

First-in-human • New P1/2 trial • Oncology • Solid Tumor

Update on the INSPIRE DUCHENNE Phase 1/2 Study of SGT-003 Microdystrophin Gene Therapy (ICNMD 2026) - "Together, these results suggest an overall favorable safety profile and a positive impact on muscle integrity as a result of SGT-003 microdystrophin expression. Additional updates on this study and the SGT-003 program will be provided at the time of presentation."

First-in-human • Gene therapy • P1/2 data • Anorexia • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Thrombocytopenia • TTN

SGT-003-301: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (clinicaltrialsregister.eu) - P2/3 | N=20 | Not yet recruiting | Sponsor: Solid Biosciences Inc.

New P2/3 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Novel Pharmacokinetic and Pharmacodynamic Effects of AAV-SLB101, Solid Biosciences’ Rationally Designed, Next-Generation Capsid (ASGCT 2026) - P1/2, P3 | "Conclusion The differentiated pharmacokinetic profile of AAV-SLB101, seen across pre- clinical and clinical studies is hypothesized to contribute to the encouraging safety and tolerability profile seen as of the January 9, 2026, cutoff date in the INSPIRE DUCHENNE trial. Further, SGT-003 has been administered with a short-term, corticosteroid-only prophylactic immunomodulatory regimen, signaling a limited inflammatory response to the vector."

PK/PD data • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Inflammation • Muscular Dystrophy

SGT-003 Gene Therapy Restores DAPC Stability and Improves Muscle Integrity in Duchenne Muscular Dystrophy (ASGCT 2026) - "The reduction in eMHC expression suggests effective interruption of the pathological degeneration- regeneration loop that drives disease progression in Duchenne. Together, these findings support robust transgene potency and functional correction at both structural and biochemical levels, underscoring the potential of SGT-003 to meaningfully alter the disease trajectory in Duchenne muscular dystrophy."

Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • TTN

Update on the INSPIRE DUCHENNE Phase 1/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy (ASGCT 2026) - "Conclusion Together, these results suggest an overall favorable safety profile and a positive impact on muscle integrity as a result of SGT-003 microdystrophin expression. Additional updates on this study and the SGT-003 program will be provided at the time of presentation."

First-in-human • Gene therapy • P1/2 data • Anorexia • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Thrombocytopenia • TTN

Solid Biosciences Announces Receipt of European Commission Orphan Drug Designation for SGT-003 for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "SGT-003 is being evaluated in two ongoing clinical trials designed to support potential regulatory authorizations in multiple countries: INSPIRE DUCHENNE, a Phase 1/2 clinical trial, and IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled clinical trial."

Orphan drug • Trial status • Duchenne Muscular Dystrophy

Gene therapy in Duchenne muscular dystrophy. (PubMed, Arch Pediatr) - "In 2023, delandistrogene moxeparvovec (Sarepta/Roche) received accelerated approval from the U.S. Food and Drug Administration for ambulatory pediatric patients, marking the first regulatory authorization of a gene therapy for DMD. By contrast, fordadistrogene movaparvovec (Pfizer) showed encouraging biomarker results but was associated with immune-mediated serious adverse events, including thrombotic microangiopathy cases and patient deaths due to acute liver failure, ultimately leading to program discontinuation. Other investigational candidates-GNT0004 (Généthon), SGT-003 (Solid Biosciences), and RGX-202 (Regenxbio)-incorporate distinct promoter designs and microdystrophin cassettes and are currently in early- to mid-phase evaluation...Gene therapy management also raises difficult economic and logistical challenges for healthcare systems. Balancing rapid patient access to potentially disease-modifying therapies with rigorous scientific and regulatory..."

Journal • Review • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Liver Failure • Muscular Dystrophy • Myositis • Pediatrics

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) (clinicaltrials.gov) - P3 | N=80 | Recruiting | Sponsor: Solid Biosciences Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov) - P1/2 | N=60 | Recruiting | Sponsor: Solid Biosciences Inc. | N=40 ➔ 60

Enrollment change • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) (clinicaltrials.gov) - P3 | N=80 | Not yet recruiting | Sponsor: Solid Biosciences Inc.

New P3 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

SGT-003: A Next-Generation Microdystrophin Gene Therapy Utilizing the Rationally Designed AAV-SLB101 Capsid (ESGCT 2025) - No abstract available

Gene therapy • Gene Therapies

Insight into the mechanism of action of AAV-SLB101, a novel muscle-tropic capsid for neuromuscular and cardiac indications (ASGCT 2025) - P1/2 | "Presently, it is utilized in Solid Bioscience's next generation gene therapy SGT-003, which is currently in a clinical study for the treatment of DMD (NCT06138639)...These studies provide further insight and characterization of the mechanism for AAVSLB101. Disease Focus of Abstract:Other Other: Neuromuscular and cardiac indications"

Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Initial Experience From the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy (ASGCT 2025) - "Preliminary biomarker data from the first 3 participants that have completed the Day 90 visit will be presented. These initial results suggest a positive safety profile of SGT-003, using only glucocorticoids for immunomodulation."

Gene therapy • Late-breaking abstract • P1/2 data • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy • Myositis • Pediatrics

AAV-SLB101, a Novel Muscle-Tropic Capsid, Increases Gene Delivery and Expression Versus AAV9 and AAVrh74, in Mouse Models of DMD and FSHD Muscle Disease (ASGCT 2025) - P1/2 | "AAV-SLB101, a rationally designed capsid with the goal of increasing muscle tropism, is used in Solid Biosciences' next generation gene therapy SGT-003, which is currently in a clinical study for the treatment of Duchenne muscular dystrophy (DMD) (NCT06138639)...In mouse models of DMD and FSHD muscle diseases, increased muscle expression of therapeutic transgenes enabled disease modulating intervention at lower doses, highlighting the superiority of AAV-SLB101 over AAV9 and AAVrh74. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Duchenne Muscular Dystrophy • Gene Therapies • DUX4 • WFDC3

Solid Biosciences to Present at the American Society of Gene and Cell Therapy’s 28th Annual Meeting (GlobeNewswire) - "Solid Biosciences Inc...will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), from May 13-17, 2025, in New Orleans, LA."

P1/2 data • Duchenne Muscular Dystrophy

Solid Biosciences to Present at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (GlobeNewswire) - "Solid Biosciences Inc...will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March 16-19, 2025."

P1/2 data • Duchenne Muscular Dystrophy

Solid Biosciences to Present at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (GlobeNewswire) - "Solid Biosciences Inc...will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003...intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March 16-19, 2025."

P1/2 data • Duchenne Muscular Dystrophy

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 (GlobeNewswire) - P1/2 | N=43 | INSPIRE DUCHENNE (NCT06138639) | Sponsor: Solid Biosciences Inc | "Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience...SGT-003 was well-tolerated in the first six participants dosed as of the data cutoff date of February 11, 2025. As of the cutoff date, all six participants have reached at least 20 days post SGT-003 treatment. Adverse events (AEs) observed after SGT-003 treatment were typical of those observed in AAV gene therapy, including nausea, vomiting, fever and transient declines in platelets in some participants. No serious adverse events (SAEs) or suspected unexpected serious adverse reactions (SUSARs) were observed...Solid expects to activate additional trial sites by the end of 2025."

P1/2 data • Duchenne Muscular Dystrophy

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov) - P1/2 | N=43 | Recruiting | Sponsor: Solid Biosciences Inc. | Trial completion date: May 2030 ➔ May 2031 | Trial primary completion date: May 2026 ➔ May 2027

Gene therapy • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Solid Biosciences Inc. | Trial completion date: Jan 2030 ➔ May 2030 | Trial primary completion date: Jan 2026 ➔ May 2026

Gene therapy • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

SGT-003, a Next Generation Microdystrophin Gene Therapy for Treatment of Duchenne Muscular Dystrophy (New Directions 2024) - No abstract available

Gene therapy • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver (ASGCT 2024) - "Among them, AAV-SLB101 has a 2-4x increase in muscle transduction and ~0.5x decrease in liver tropism in comparison to AAV9 in mice, both DMDmdx and wild type, and non-human primates. This capsid is being used in Solid Biosciences' SGT-003 program for the treatment of DMD that recently had an IND cleared by the US FDA...Through the combination of liver detargeting and muscle targeting modifications in wild type capsids the resulting capsids maintained or improved cardiac and musculoskeletal transduction while detargeting the liver. These precisely engineered capsids could lead to gene therapy vectors with high efficiency, efficacy, and safety profiles for treating cardiac and musculoskeletal indications, which require a systemic delivery approach."

Gene Therapies • Hepatology • Liver Failure • Musculoskeletal Diseases

Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy (ASGCT 2024) - "Improvements in muscle pathology were also observed in response to microdystrophin expression. Overall, these data provided proof of concept for the use of SGT-003 in the treatment of DMD and supported initiation of a Phase I/II clinical study in boys with DMD."

Gene therapy • Preclinical • Cardiovascular • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy