SGT-003 / Solid BioSci - LARVOL DELTA

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

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 (GlobeNewswire) - "Solid Biosciences Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate....The planned Phase 1/2 trial, INSPIRE Duchenne, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg....We anticipate providing an initial safety update for the first three to four patients enrolled in the INSPIRE Duchenne trial in mid-2024, and we anticipate providing initial expression and functional data from those patients in the fourth quarter of 2024."

FDA event • New P1/2 trial • P1/2 data • Duchenne Muscular Dystrophy • 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. | Not yet recruiting ➔ Recruiting

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

Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 (GlobeNewswire) - "Solid Biosciences Inc...announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate....The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003 and expects to commence patient screening shortly thereafter. Patient dosing in the trial is expected to commence in mid-to-late first quarter of 2024."

New P1/2 trial • Orphan drug • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Solid Biosciences Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Gene Therapy SGT-003 (GlobeNewswire) - "Solid Biosciences Inc....announced that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate."

Fast track designation • Duchenne Muscular Dystrophy • Muscular Dystrophy