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Scientific Publications & Presentations

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ASGCT 2026
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
Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT 212) for the Treatment of Friedreich’s Ataxia
ASGCT 2026
Development and Implementation of a High Yield and Scalable Manufacturing Process for Adeno Associated Virus (AAV) Production
ASGCT 2026
Boosting rAAV Yield in HEK293 Cells Through Multi Mechanism Strategies
ASGCT 2026
Utilizing Machine Learning and Mechanistic Understanding to Appreciate the Impact of pH, Dissolved Oxygen, and pCO₂ on Upstream AAV Yield and Product Quality
ASGCT 2026
Novel Pharmacokinetic Effects of POLARIS-101™ (AAV-SLB101), Solid Biosciences’ Rationally Designed, Next Generation Capsid
ASGCT 2026
SGT 003 Gene Therapy Restores DAPC Stability and Improves Muscle Integrity in Duchenne Muscular Dystrophy
ASGCT 2026
A Novel AAV Gene Therapy Strategy to Correct Calcium Dysregulation in Catecholaminergic Polymorphic Ventricular Tachycardia
ASGCT 2026
Systemic Delivery of Human TNNT2 Gene Therapy Using the Novel Capsid POLARIS-101TM (AAV-SLB101) Improves Cardiac Function in the TNNT2 R141W Knock in Mouse Model of Dilated Cardiomyopathy
ASGCT 2026
Systemic Administration of a Rationally Engineered Vector (POLARIS-101TM [AAV-SLB101]) Leads to Successful Transduction of Non Human Primate Tissues in the Presence of Neutralizing Antibodies
ASGCT 2026
Lower POLARIS-101™ (AAV-SLB101) Cross Reactive Antibodies in Elevidys Treated Patients
ASGCT 2026
Small Molecule Induced G2/M Arrest Boosts Recombinant AAV Production and Preserves High Quality
ASGCT 2026
Impact of Genome Size and Sequence Composition on AAV Vector Genome Integrity
ASGCT 2026
Connecting Changes in AAV Capsid Protein Charge Species to In Vitro Protein Expression
ASGCT 2026
Impact of Full vs. Empty AAV Capsids on Vector Potency
ASGCT 2026
Identifying Sources of Library Preparation Artifacts in AAV Vectors with SMRT Sequencing
MDA 2026
Update on the INSPIRE DUCHENNE Phase 1/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy
MDA 2026
Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia
MDA 2026
Novel Pharmacokinetic Effects of POLARIS-101™, Solid Biosciences’ Rationally Designed, Next-Generation Capsid
MDA 2026
SGT-003 Demonstrates High Cardiac Tropism and Positive Preliminary Clinical Findings Using the Next-Generation Muscle-Tropic Capsid
MDA 2026
SGT-003 Gene Therapy Stabilizes the DAPC and Improves Muscle Integrity in Duchenne Muscular Dystrophy
MDA 2026
Positive Preliminary Safety and Liver Toxicity Profile Using SGT-003, Solid Biosciences’ Next-Generation Investigational Gene Therapy for Duchenne Muscular Dystrophy
CVCT
Cardiovascular Clinical Trialist Forum: Cardiac Gene Treatment
CVCT
AAV-SLB101: A Next-Generation Rationally Designed Capsid Demonstrates Highly Potent Cardiac Tropism and Initial Clinical Safety
CVCT
AAV-SLB101: A Next-Generation Rationally Designed Capsid Demonstrates Highly Potent Cardiac Tropism and Initial Clinical Safety (Presentation)
Society for Neuroscience Annual Meeting
Efficacy and Safety of a Novel AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia
AHA Scientific Sessions
Efficacy and safety of a novel AAV FXN gene therapy (SGT-212) for the treatment of Friedreich’s ataxia
WMS
Comprehensive Evaluation of Muscle Integrity Biomarkers to Assess Therapeutic Efficacy in Duchenne Muscular Dystrophy
WMS
Update on INSPIRE DUCHENNE: a phase 1/2 study of SGT-003, a next-generation microdystrophin gene therapy for Duchenne muscular dystrophy
ESGCT
Successful Cardiac Gene Transfer With a Rationally Designed AAV Capsid in the Presence of Anti-AAV Neutralizing Antibodies
ESGCT
EV-AAV Enhances Transgene Expression Via Partial Protection from Neutralizing Antibodies In Vitro
ESGCT
Correction of CPVT-Related Electrophysiological Abnormalities by CASQ2 Overexpression
ESGCT
SGT-003: A Next-Generation Microdystrophin Gene Therapy Utilizing the Rationally Designed AAV-SLB101 Capsid
NMSG
AAV-SLB101 in Duchenne Muscular Dystrophy: Nonclinical Safety, Characterization of Efficacy, and Preliminary Clinical Insights
NMSG
Update on INSPIRE DUCHENNE: A Phase 1/2 Study of SGT-003, an Investigational Next-Generation Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
NMSG
SGT-003: Initial Safety Evaluation of a Next-Generation Investigational Gene Therapy for Duchenne Muscular Dystrophy
ASGCT 2025
Initial Experience From the INSPIRE DUCHENNE Phase 1/2 Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
ASGCT 2025
Insight Into the Mechanism of Action of AAV-SLB101, a Novel Muscle-Tropic Capsid for Neuromuscular and Cardiac Indications
ASGCT 2025
Potential for AAV-SLB101–Mediated Gene Transfer Treatment in the Context of Natural Seropositivity and After an AAVrh74 Treatment
ASGCT 2025
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
Full-Length Transgene Quantification Utilizing NanoMosaic Tessie Technology
ASGCT 2025
Automation of AAV Capsid ELISA on Tecan Fluent
ASGCT 2025
Capillary Isoelectric Focusing (cIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on Their Transduction Efficiency
PPMD 2024
A Next Generation Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
ASGCT 2024
Novel Mechanism to Increase AAV Yield through Blocking AAV Transduction of Manufacturing HEK293 Cells During AAV Production
ASGCT 2024
Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver
ASGCT 2024
Designing Therapeutic Recombinant AAV Vectors Using In Silico Vector Modeling
ASGCT 2024
Identification of an AAV Affinity Chromatography Elution Buffer that Maximizes Product Recovery and Minimizes Product Degradation
ASGCT 2024
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
Increasing Quality and Productivity with Dual Transfection (DT) for AAV Production
ASGCT 2024
High-Throughput Workflows to Accelerate Development of Chromatographic Purification of rAAV Viral Vectors
2025 MDA Clinical & Scientific Conference
Initial Experience from the INSPIRE DUCHENNE Phase 1/2 Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Frontiers in Physiology
Prolonged voluntary wheel running reveals unique adaptations in mdx mice treated with microdystrophin constructs ± the nNOS-binding site
ASGCT 2023
Novel AAV Capsid Identification and Characterization for Neuromuscular and Cardiac Indications
ASGCT 2023
Genomic Characterization of AAV Products Using Multiplex ddPCR and Nanopore Sequencing
ASGCT 2023
Development and Qualification of Multiplexed ddPCR Assay to Evaluate DNA Integrity
ASGCT 2023
Efficacy and Safety of a Novel AAV FXN Gene Therapy (AVB-202) for the Treatment of Friedreich’s Ataxia
ASGCT 2023
Characterization of Genomic Heterogeneity in rAAV Preparations Using Short- and Long-Read Next Generation Sequencing
ASGCT 2023
Characterization of Factors that Influence the Yield and Quality of rAAV Produced Using HSV Co-Infection
MDA 2023
IGNITE DMD One-Year Post Treatment Study Analysis
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