Scientific & Medical Presentations

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 CapsidsPresented
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 EditingPresented
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