SGT-001 / Solid BioSci - LARVOL DELTA

In vitro and clinical assessments of muscle specific promoters in driving gene expression and long-term cardiac efficacy in Duchenne muscular dystrophy (ASGCT 2025) - P1, P1/2 | "Three of these products include zildistrogene varoparvovec (SGT-001), an AAV9 vector containing a CK8 promoter, fordaditrogene movaparvovec (PF-06939926), an AAV9 vector containing an hCK promoter, and delandistrogene-moxeparvovec (SRP-9001, ElevidysÒ), an AAVrh74 vector containing a MHCK7 promoter. This two-part study illustrated the Desmin promoter was superior to the others in transfecting skeletal muscle and cardiac function was preserved over time in patients treated with gene therapy. Further studies are needed to test efficacy of each promoter in human cardiomyocytes and additional long term follow up is needed to understand the effects of DMD gene therapy are sustained in cardiac muscle."

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

Hepatic-Restricted Microdystrophin Expression Induces Immune Tolerance and Enhances Transgene Expression in Dystrophin-Deficient Mdx Mice (ASGCT 2025) - "µDys9.2 differs from PF-06939926 and SRP-9001 by including the nNOS binding domain, and from SGT-001 by including R3 and excluding R23...Group 1 received the anti-CD20 regimen, followed by AAV-Des-µDys9.2 i.v. administration (1 × 10¹⁴ vg/kg) at eight weeks of age, along with continuous sirolimus dosing until week 12... Hepatic-restricted and nonhepatic µDys9.2 delivery, combined with IMS, enhances transgene expression and immune tolerance in mdx mice, improving efficacy and safety. Disease Focus of Abstract:Rare Diseases"

Preclinical • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Muscular Dystrophy • Rare Diseases • Respiratory Diseases • FOXP3 • IL2RA

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2 | N=12 | Active, not recruiting | Sponsor: Solid Biosciences Inc. | Trial primary completion date: Oct 2022 ➔ Oct 2026

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

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2 | N=12 | Active, not recruiting | Sponsor: Solid Biosciences Inc. | Trial completion date: Dec 2027 ➔ Oct 2026

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

Longitudinal changes of Anti-AAV9 and Anti-AAVrh74 antibodies in treated and untreated patients (MDA 2024) - "Subjects received SGT-001 (N=7), Zolgensma (N=7), PF-06939926 (N=2), AT132 (N=1), LYS-SAF302 (N=1), PGTC PD-AAV004 (N=2), all AAV9 products... 169 untreated subjects (115 male) with 1 plasma/serum sample for analysis were included. Median age was 13 (3-37). Of these, 92 (54%) were seropositive for Anti-AAV9 IgG at initial collection."

Clinical • CNS Disorders • Gene Therapies • Immunology

Evaluation of long-term cardiac MRI outcome following micro-dystrophin gene therapy in subjects with Duchenne muscular dystrophy receiving SGT-001 (MDA 2024) - "This is the first study that describes longitudinal cMRI findings in DMD subjects that have received micro-dystrophin gene therapy. We observed that cardiac function was preserved as the individuals age with expected worsening of DMD-related cardiomyopathy. Further studies are needed to better understand the effects of the DMD gene therapy in the heart and carefully quantify the extent of cardiac gene transfer."

Clinical • Gene therapy • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2 | N=16 | Active, not recruiting | Sponsor: Solid Biosciences Inc. | Trial completion date: Dec 2028 ➔ Dec 2027 | Trial primary completion date: Dec 2023 ➔ Dec 2026

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

DMD patients treated by SGT-001 Microdystrophin Gene Therapy improve in the objective endpoint of spontaneous walking velocity (WMS 2022) - "SGT-001 treated subjects showed an average improvement in SV95C at all post-treatment timepoints, with positive results observed in both dose groups. Together with previously presented data, these results provide additional evidence that patients treated with SGT-001 may be experiencing a benefit to motor function and a clinical course that diverges from untreated patients."

Clinical • Late-breaking abstract • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

IGNITE DMD phase I/II study of SGT-001 microdystrophin gene therapy for DMD: Long-term outcomes and expression update (WMS 2022) - "Previously presented data from earlier dosed patients at up to 2 years post-dosing suggest the durability of these effects. Together, these preliminary data continue to suggest a positive benefit-risk profile for SGT-001 in DMD patients."

P1/2 data • Duchenne Muscular Dystrophy • Gene Therapies • Muscular Dystrophy

IGNITE DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for DMD: 2-Year Outcomes Update (ICNMD 2022) - No abstract available

P1/2 data • Gene Therapies

IGNITE DMD Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: Long-Term Outcomes and Biomarker Update (ASGCT 2022) - "Interim results from more recently dosed patients will also be discussed. These preliminary data continue to suggest a positive benefit-risk profile for SGT-001 warranting continued evaluation for the treatment of DMD."

Biomarker • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Pain • Pediatrics • Thrombocytopenia

Systematic Literature Review Assessing the Clinical Outcomes of Adeno-Associated Virus (AAV) Gene Transfer Therapies in Duchenne Muscular Dystrophy (DMD) (ISPOR 2022) - "RESULTS : This SLR identified 6 publications (one journal article and five conference abstracts) reporting results from two unique therapies SGT-001 and rAAVrh74.MHCK7. micro-dystrophin (SRP-9001)...CONCLUSIONS : Currently, clinical trials for gene transfer therapies in DMD patients are in phase I or II. Preliminary results suggest that these therapies are efficient, safe, and well tolerated."

Clinical • Clinical data • Review • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Infectious Disease • Muscular Dystrophy

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2 | N=16 | Active, not recruiting | Sponsor: Solid Biosciences, LLC | Recruiting ➔ Active, not recruiting

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

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: Solid Biosciences, LLC; Trial completion date: Apr 2027 ➔ Dec 2028; Trial primary completion date: Apr 2022 ➔ Dec 2023

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

SGT-001: AAV Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy (New Directions 2021) - No abstract available

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

[VIRTUAL] IGNITE-DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy (ASGCT 2021) - "In addition to protein expression and biomarker results, positive results were observed in functional and quality of life assessments at 1 year post-treatment and will be described. These data in totality continue to support the ongoing investigation of SGT-001 as a transformative therapy for DMD."

P1/2 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hematological Disorders • Inflammation • Muscular Dystrophy • Thrombocytopenia

[VIRTUAL] IGNITE-DMD: Phase I/II Ascending Dose Study of Single IV Infusion of SGT-001 Microdystrophin Gene Therapy for DMD-One Year Efficacy and Safety Results (MDA 2021) - "Additional biomarker and outcomes will also be reported. These preliminary findings support the continued investigation of SGT-001 as a potential therapy for DMD."

Clinical • P1/2 data • Acute Kidney Injury • Gene Therapies • Hematological Disorders • Hepatology • Nephrology • Renal Disease • Thrombocytopenia

[VIRTUAL] IGNITE-DMD: Phase I/II Ascending Dose Study of Single IV Infusion of SGT-001 Microdystrophin Gene Therapy for DMD-One Year Efficacy and Safety Results (MDA 2021) - No abstract available

Clinical • P1/2 data • Gene Therapies

Efficacy and Safety Data from Solid Biosciences’ Ongoing IGNITE DMD Phase I/II Clinical Trial to be Presented at the 2021 MDA Clinical & Scientific Conference (GlobeNewswire) - "Company to host external key opinion leaders at a symposium to discuss Real World Outcomes Measures in Duchenne Muscular Dystrophy during the conference; Company to host conference call to discuss the interim one-year clinical trial data and fourth quarter and full year 2020 financial results on Monday, March 15 at 4:30 PM ET; Solid Biosciences Inc....announced that one-year efficacy and safety data from the ongoing IGNITE-DMD Phase I/II study of SGT-001 microdystrophin gene therapy in patients with Duchenne muscular dystrophy (Duchenne) will be presented at the 2021 MDA Virtual Clinical & Scientific Conference."

P1/2 data • Duchenne Muscular Dystrophy

Form 8-K Solid Biosciences Inc. For: Jan 11 (Streetinsider.com) - "On January 14, 2021, Solid Biosciences Inc....will present at the 39th Annual J.P. Morgan Healthcare Conference. The Company’s presentation will provide updates regarding anticipated upcoming milestones for its Phase I/II clinical trial called IGNITE DMD, including that the Company plans to present 12-month safety and efficacy data for all patients dosed to date in IGNITE DMD in the first quarter of 2021 and that the Company anticipates presenting 90-day biopsy data for additional patients dosed in IGNITE DMD in the second half of 2021."

P1/2 data • Duchenne Muscular Dystrophy

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: Solid Biosciences, LLC; Active, not recruiting ➔ Recruiting

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

IGNITE DMD: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (clinicaltrials.gov) - P1/2; N=16; Active, not recruiting; Sponsor: Solid Biosciences, LLC; Suspended ➔ Active, not recruiting; Trial completion date: Mar 2024 ➔ Apr 2027; Trial primary completion date: Mar 2023 ➔ Apr 2022

Clinical • Enrollment closed • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy (GlobeNewswire) - "Ultragenyx...today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne....Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy...Solid expects to dose the next patient in the IGNITE DMD clinical trial, using SGT-001 produced using its improved HSV manufacturing process, in the first quarter of 2021."

Enrollment status • Licensing / partnership • Becker Muscular Dystrophy • Duchenne Muscular Dystrophy

Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial (GlobeNewswire) - "Solid Biosciences...today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial...Based on the Company’s response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions...'We are working diligently to complete all activities necessary to resume dosing, which we expect to occur in the first quarter of 2021.'"

FDA event • Duchenne Muscular Dystrophy

Solid Biosciences Provides Update Regarding the IGNITE DMD Phase I/II Clinical Hold (Solid Biosciences Press Release) - "Solid Biosciences...today announced that it received written communication from the U.S. Food and Drug Administration (FDA) that the Company’s IGNITE DMD Phase I/II clinical trial remains on clinical hold...Today, the FDA responded by maintaining the clinical hold and requesting further manufacturing information, updated safety and efficacy data for all patients dosed, and providing direction on total viral load to be administered per patient."

FDA event • Duchenne Muscular Dystrophy