Sangamo Therapeutics to Present Neurology Pipeline Advances at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
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Scientific & Medical Presentations
Neurology Conference Presentations Hub
WORLDSymposium 2025
Presentation: Isaralgagene Civaparvovec (ST-920) Gene Therapy in Adults with Fabry Disease: Updated Results from an Ongoing Phase 1/2 Study (STAAR)
WORLDSymposium 2025
Poster: Isaralgagene Civaparvovec (ST-920) Gene Therapy in Adults with Fabry Disease: Updated Results from an Ongoing Phase 1/2 Study (STAAR)
ASH 2024
Efficacy and Safety of Giroctocogene Fitelparvovec in Adults With Moderately Severe to Severe Hemophilia A: Primary Analysis Results From the Phase 3 AFFINE Gene Therapy Trial
Prion 2024
Presentation: Single Intravenous Treatment with Zinc Finger Repressor Leads to Brainwide Reduction of Prion in Nonhuman Primates and Significantly Prolongs Survival in the RML Mouse Model
ASGCT 2024
Presentation: A Highly Potent Engineered AAV Capsid, STAC-150, Enables High-Throughput AAV Production and Arrayed Epigenetic Regulator Screening Directly in Cultured Neurons
ASGCT 2024
Presentation: Directed Evolution of Bxb1 for the Development of Modular Integrases (MINTS)
ASGCT 2024
Presentation: Identification and Characterization of STAC-BBB, an Engineered AAV Capsid That Exhibits Widespread Transduction of the Central Nervous System in Cynomolgus Macaques
ASGCT 2024
Poster: Site-directed integration of large DNA sequences into endogenous sites in the human genome using engineered Modular Integrases (MINTS)
ASGCT 2024
Poster: Highly Specific Zinc Finger Proteins with Synthetic Target Sites Enable Self-Regulated Expression of Dosage-Sensitive Transgenes
ASGCT 2024
Poster: Zinc Finger Mediated Repression and Replacement of MFN2 Leads to the Rescue of Cellular Disease Phenotype in CMT2A Patient-Derived Cells
ASGCT 2024
Poster: Process and Formulation Development for a Novel Blood-Brain Barrier Penetrant AAV Capsid
ASGCT 2024
Poster: Unraveling Impact of Manufacturing Process-Related Stresses on AAV Stability, Aggregation, and DNA Release
ASGCT 2024
Poster: Development of Blood-Brain Barrier Penetrant AAVs through Receptor-Targeted Capsid Engineering
ASGCT 2024
Poster: Optimal Drug Product Presentation and Container Closure Selection for AAV-Based Genomic Medicines
ASGCT 2024
Poster: Epigenetic Regulation of Human Prion Expression as a Potential One-Time Treatment for Prion Disease
ASGCT 2024
Poster: Development of a Robust Zinc Finger Activation Platform for Treatment of Neurological Disorders
ASGCT 2024
Poster: Shank3 Gene Activation Mediated by Zinc Finger Transcriptional Activators (ZFA) as a Therapeutic Approach for Phelan-McDermid Syndrome
ASGCT 2024
Poster: PMP22 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for CMT1A
ASGCT 2024
Poster: SOD1 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for SOD1-Mediated ALS
ASGCT 2024
Poster: Widespread CNS Tau Knockdown for the Potential Treatment of Alzheimer’s Disease and other Tauopathies
ASGCT 2024
Poster: UBE3A Gene Activation Mediated by Zinc Finger Activators (ZFAs) as a Therapeutic Approach for Angelman Syndrome
ASGCT 2024
Poster: SNCA Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for Parkinson’s Disease
ASGCT 2024
Poster: A Zinc Finger Activator Platform to Restore Normal Gene & Protein Expression in Cellular Models of Dravet Syndrome
ASGCT 2024
Poster: Restoration of Normal Gene and Protein Expression in Mouse and Human Disease Models of SCN2A Haploinsufficiency Using Zinc Finger Activators
WORLDSymposium 2024
Presentation: Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
WORLDSymposium 2024
Poster: Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
WORLDSymposium 2024
Presentation: A 3-month gene therapy single-dose IV administration pharmacology and safety study with ST-920 (isaralgagene civaparvovec) for Fabry disease in mice
WORLDSymposium 2024
Poster: A 3-month gene therapy single-dose IV administration pharmacology and safety study with ST-920 (isaralgagene civaparvovec) for Fabry disease in mice
WORLDSymposium 2024
Poster: Anti-AAV6 antibody assay for patient enrollment supporting ST-920 phase 1/2 study for Fabry disease
ASH 2023
Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults With Severe Hemophilia A
CRL Biotechnology Symposium 2023
Preclinical Development of a Zinc Finger Transcriptional Repressor Targeting the SCN9A Gene as a Novel Therapy for Peripheral Neuropathic Pain
American College of Toxicology 2023
Preclinical Development of a Novel Gene Therapy for the Treatment of Peripheral Neuropathic Pain Through Zinc Finger Repressor- Mediated Repression of the SCN9A Gene
Annual Pain Therapeutics Summit 2023
Zinc finger mediated epigenetic repression of SCN9A gene as a therapeutic approach for painful peripheral neuropathies
ESGCT 2023
Multiplex targeting of immune checkpoints with compact zinc finger repressors to improve anti-tumor activity of T cells
ESGCT 2023
Zinc Finger Activators restore normal gene and protein expression in a mouse model of SCN2A haploinsufficiency
ESGCT 2023
Shank3 Gene Activation Mediated by Zinc Finger Activators (ZF-As) as a Therapeutic Approach for Phelan-McDermid Syndrome
ESGCT 2023
Multimerization of Chimeric Antigen Receptor (CAR) binding domains: A solution to assess tissue specificity of low to medium affinity scFv
Prion 2023
Neuron-Specific Expression of Zinc-Finger Repressors Mediate Widespread Prion Reduction in the Brain for the Potential Treatment of Prion Disease
ASGCT 2023
Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies
ASGCT 2023
Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model
ASGCT 2023
Engineered Zinc Finger Transcriptional Regulators Enable Robust and Reliable Epigenetic Regulation in the Mouse Brain
ASGCT 2023
A Robust and Flexible Baculovirus-Insect Cell System for AAV Vector Production with Improved Yield, Capsid Ratios and Potency
ASGCT 2023
Overcoming the Effect of Previous Enzyme Replacement Therapy on the Detection of Anti-Transgene Protein Antibodies After Treatment with Gene Therapy
ASGCT 2023
Gene Activation Mediated by Zinc Finger Transcriptional Regulators (ZF-TRs) as a Therapeutic Approach for CNS Disorders
ASGCT 2023
Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
ASGCT 2023
Strategic Formulation Development for AAV Delivered Gene Therapies – a Case Study
ASGCT 2023
Development of a Competitive Ligand-Binding Assay to Detect Neutralizing Antibodies Against Chimeric Antigen Receptor of Regulatory T Cells
ASGCT 2023
Cell-Type Specific Reduction of Prion Expression in Neurons and Astrocytes Using Engineered Zinc Finger Transcriptional Regulators
ASGCT 2023
Intercellular Zinc Finger Protein Delivery for Cross-Corrective Epigenetic Regulation in the CNS
ASGCT 2023
Fitness Maturation of Engineered AAV Capsids STAC-102 and STAC-103 Enhances Central Nervous System Transduction after CSF Administration in Cynomolgus Macaques