Plenary Session: Advances in Gene Therapies: From Ex-Vivo to In-Vivo
Friday, September 8, 2023 03:00 PM - 04:30 PM
Grand Ballroom J-K
Chair
Sandeep Soni, MD, CRISPR (USA), USA
Speakers
Matthew Porteus, MD, PhD, Stanford School of Medicine, USA – Homology Directed Repair in Hematopoietic Stem Cells: Continued Iterative
David Lebwohl, MD, Intellia Therapeutics, Inc., USA – Initial successes with CRISPR-based in vivo genome editing for treatment of patients with severe illness
Shengdar Tsai, PhD, St. Jude Children's Research Hospital, USA – Understanding fundamental genome-wide activities of CRISPR genome editors
This session will focus on the recent advances, viral and non-viral delivery platforms, challenges and regulatory requirements for gene therapies, including:
Session Objectives
Sandeep Soni, MD, CRISPR (USA), USA
Speakers
Matthew Porteus, MD, PhD, Stanford School of Medicine, USA – Homology Directed Repair in Hematopoietic Stem Cells: Continued Iterative
David Lebwohl, MD, Intellia Therapeutics, Inc., USA – Initial successes with CRISPR-based in vivo genome editing for treatment of patients with severe illness
Shengdar Tsai, PhD, St. Jude Children's Research Hospital, USA – Understanding fundamental genome-wide activities of CRISPR genome editors
This session will focus on the recent advances, viral and non-viral delivery platforms, challenges and regulatory requirements for gene therapies, including:
- Ex-vivo Crispr-Cas9 gene-editing techniques in hematopoietic stem cells
- In-vivo gene-editing using unique lipid nano-particle delivery of Crispr-Cas9
- Clinical applications of ex-vivo and in vivo gene editing
- Analysis of ‘’off-target’’ activity in gene-editing and regulatory implications.
Session Objectives
- To describe ex-vivo Crispr-Cas9 gene-editing in hematopoietic stem cells
- To provide an overview of in-vivo gene-editing using unique lipid nano-particle delivery of Crispr-Cas9 To present clinical applications and early clinical results of both the platforms
- To discuss analysis of ‘’off-target’’ activity in gene-editing and it’s regulatory aspects.
Sandeep Soni MD
Executive Director, Clinical Development and Assoc. Prof of Pediatrics. UCSF
CRISPR (USA)
Chair
Executive Director, Clinical Development and Assoc. Prof of Pediatrics. UCSF
CRISPR (USA)
Chair
Shengdar Tsai PhD
Associate Member, Department of Hematology
St. Jude Children's Research Hospital
Speaker
Associate Member, Department of Hematology
St. Jude Children's Research Hospital
Speaker