News & Media:Scientific Publications & Presentations
- Efficacy and safety of a novel AAV FXN gene therapy (AVB-202) for the treatment of Friedreich’s ataxia, May 19, 2023
- Characterization of factors that influence the yield and quality of rAAV produced using HSV co-infection, May 19, 2023
- Characterization of Genomic Heterogeneity in rAAV Preparations Using Short- and Long-Read Next Generation Sequencing, May 19, 2023
- Development and Qualification of Multiplexed ddPCR Assay to Evaluate DNA Integrity, May 19, 2023
- Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications, May 19, 2023
- Genomic Characterization of AAV Products using Multiplex ddPCR and Nanopore Sequencing, May 19, 2023
- IGNITE DMD Phase I/II Study of Microdystrophin Gene Therapy for DMD: One-Year Post-Treatment Data Presented at Muscular Dystrophy Association Clinical and Scientific Conference, March 22, 2023
- Clinical Potential of Microdystrophin as a Surrogate Endpoint, December 13, 2022
- DMD Patients Treated by SGT-001 Microdystrophin Gene Therapy Improve in the Objective Endpoint of Spontaneous Walking Velocity, October 18, 2022
- IGNITE DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for DMD: Long-Term Outcomes and Expression Update, October 18, 2022
- IGNITE DMD Study of SGT-001: Microdystrophin Gene Therapy for DMD: Long-Term Outcomes and Biomarker Update Presented at ASGCT 25th Annual Meeting, May 24, 2022
- IGNITE DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for DMD: 2-Year Outcomes Update, March 21, 2022
- Solid Biosciences Reports Additional Pulmonary Function Results from the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001 at the Child Neurology Society 50th Annual Meeting, September 30, 2021
- Solid Biosciences Reports 1.5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001 at World Muscle Society 2021 Congress, September 23, 2021
- Data from Studies Characterizing Novel AAV Vectors Presented at ASGCT 23rd Annual Meeting, May 13, 2020Solid Biosciences presented data from studies characterizing novel adeno-associated (AAV) vectors engineered for muscle gene delivery in a poster session.
- Data from SGT-001 Microdystrophin Gene Therapy Program Presented at ASGCT 23rd Annual Meeting, May 13, 2020Solid Biosciences presented clinical biomarker data for SGT-001 and provides data on expression and co-localization of microdystrophin and dystrophin associated proteins in patients in the IGNITE DMD trial.
- Membrane recruitment of nNOSµ in microdystrophin gene transfer to enhance durability, September 5, 2019This review article in the journal Neuromuscular Disorders summarizes the evidence supporting the function of microdystrophin-associated neuronal nitric oxide synthase (nNOS) and its potential utility in Duchenne muscular dystrophy (DMD) gene therapy.
- PPMD 2019 Annual Conference, June 28, 2019Solid’s Vice President of Clinical R&D, Genevieve A. Laforet, MD, Ph.D., presented an overview of the SGT-001 microdystrophin gene transfer program at the Parent Project Muscular Dystrophy (PPMD) 2019 Annual Conference in Orlando, Florida.
- 22nd Annual Meeting of The American Society of Gene and Cell Therapy, May 1, 2019Solid Biosciences presented new preclinical data from its next generation gene therapy program that focuses on the Company’s efforts to screen for potential novel promoters.
- 17th Annual International Conference For Duchenne and Becker Muscular Dystrophy, February 25, 2019Solid’s Chief Scientific Officer, Carl Morris, Ph.D., recently presented an overview of the SGT-001 microdystrophin gene transfer program at the Duchenne Parent Project Onlus 17th Annual International Conference for Duchenne and Becker Muscular Dystrophy in Rome.
- Development of Novel Micro-dystrophins with Enhanced Functionality, February 1, 2019In this paper, Solid collaborator and Scientific Advisory Board Chair Dr. Jeffrey Chamberlain and other researchers showed preclinical data that highlight the unique attributes of the SGT-001 transgene.
- 23rd International Annual Congress of the World Muscle Society, October 6, 2018Solid Biosciences presented data on kinematic biomarkers that may help identify the progression of the disease in patients with Duchenne muscular dystrophy.
- 21st Annual Meeting of The American Society of Gene and Cell Therapy, May 18, 2018Solid Biosciences presented preclinical data that demonstrate the potential of SGT-001 as a treatment candidate for Duchenne muscular dystrophy. Additionally, the Company presented data that support the development of novel promoters and capsids as part of its next-generation gene therapy discovery efforts.
- A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy, July 27, 2017In this paper, Solid’s Scientific Advisory Board member, Dongsheng Duan and a team of researchers at the University of Missouri offered preclinical data in a symptomatic mouse model to support the further development of nNOS-binding five-repeat microdystrophin gene therapy constructs for DMD.