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Scientific & Medical Presentations
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
ASGCT 2023
Process and Platform Development for Production and Purification of CNS-Tropic Engineered AAV Capsids
ASGCT 2023
Zinc Finger-Transcriptional Repressors Against Immune Checkpoint Molecules to Improve Anti-Tumor Activity of Gene-Modified T Cells
WORLDSymposium 2023
Poster: STAAR, a Phase 1/2 study of isaralgagene civaparvovec (ST 920) gene therapy in adults with Fabry disease
WORLDSymposium 2023
Presentation: STAAR, a Phase 1/2 study of isaralgagene civaparvovec (ST 920) gene therapy in adults with Fabry disease
ASH 2022
Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults With Severe Hemophilia A
ASH 2022
Interim Safety and Efficacy Results From a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)
Prion 2022
ZF-transcription factors for Prion Disease
NORD 2022
Preliminary results of STAAR, a Phase I/2 study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease and long-term follow-up
ESGCT 2022
Preliminary results of STAAR, a Phase 1/2 study of isaralgagene civaparvovec (ST 920) gene therapy in adults with Fabry disease and long term follow up
ESGCT 2022
Engineering of Allogeneic Regulatory T Cells Expressing a Chimeric Antigen Receptor (Allo-CAR-Tregs) Using Zinc Finger Nuclease/AAV6-Mediated Editing
ESGCT 2022
Engineering of Factor IX-secreting B cells using ZF-Nuclease / AAV6 editing technology in a GMP-compatible medium
SSIEM 2022
Preliminary results of STAAR, a Phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease and long-term follow-up
ASGCT 2022
Engineered AAV Capsids Exhibit Improved Transduction of the Central Nervous System After CSF Administration in Adult Cynomolgus Macaques
ASGCT 2022
A Compact Zinc Finger Architecture for Highly Efficient Base Editing in Human Cells
ASGCT 2022
STEADFAST: A First-in-Human Study Assessing HLA-A*02-Chimeric Antigen Receptor Regulatory T Cells in Renal Transplantation
ASGCT 2022
Evaluation of a Human Neurovascular Model to Complement a Parallel Non-human Primate Selection for Blood–Brain Barrier Penetrant AAV Capsids
ASGCT 2022
Zinc Finger Nuclease/AAV6-Mediated Factor IX(FIX) Insertion Into B Cells Results in Sustained Therapeutic FIX Levels In Vivo After Using a GMP-Like Differentiation Culture System
ASGCT 2022
Genome Orthogonal Zinc Finger Proteins for the Development of Genomic Medicines
ASGCT 2022
Engineering of Allogeneic Regulatory T Cells Expressing a Chimeric Antigen Receptor(Allo-CAR-Tregs) Using Zinc Finger Nuclease/AAV6-Mediated Editing
ASGCT 2022
Durable, Multiplexed Cell Engineering Using Zinc Finger–Guided Transcriptional Regulators Delivered via a Single Viral Particle
WORLDSymposium 2022
Preliminary Results of the STAAR Study, a Phase I/II Study of Isaralgagene Civaparvovec (ST-920) Gene Therapy in Adults With Fabry Disease
ASH 2021
Preliminary Safety and Efficacy Results from PRECIZN-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)
ASH 2021
Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF 07055480/SB 525) Gene Therapy in Adults With Severe Hemophilia A
ASH 2021
3974 Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
ESGCT 2021
IL23R-CAR-Tregs – A Novel Approach to Treat Crohn’s Disease (CD) Patients
ESOT 2021
Design of STEADFAST Study Evaluating TX200-TR101: A Chimeric Antigen Receptor (CAR) T Regulatory Cell Therapy for Living Donor Renal Transplant Recipients
ASGCT 2021
Selective Repression of C9ORF72 Repeat Expansion-Containing Sense and Antisense Transcripts for the Treatment of ALS
ASGCT 2021
Zinc Finger Protein Transcription Factors Targeting Alpha-synuclein – A Promising Therapeutic Strategy for Parkinson’s Disease
AD/PD 2021
Zinc Finger Protein Transcription Factor-Mediated Repression of Alpha-Synuclein Expression as a Therapeutic Approach for Parkinson’s Disease
WORLDSymposium 2021
A Phase I/II Multicenter Gene Therapy Clinical Study for Fabry Disease