Webinars » GenScript Webinar Series: Gene Editing & Delivery Tools

GenScript Webinar Series: Gene Editing & Delivery Tools

This Webinar Series will feature Dr. John Zuris, Dr. Samantha Yost, and Dr. Lili Yang – three leaders in the life sciences from academia and industry – who are leveraging the strengths of synthetic biology technologies to develop life-changing therapies. Synthetic biology continues to revolutionize life science research, advancing gene and cell therapies and enabling the efficient development of other invaluable biotherapeutics for various human diseases. Join GenScript in this series of Webinars!

In this webinar series, you will learn about:

New CRISPR-based technologies for allogeneic T cell engineering

High efficiency multiplex gene editing with Cas12a

Unbiased analysis of AAV tissue tropism with whole mouse-body imaging 

Engineering and differentiating HSCs to produce allogeneic iNKT cell therapies 

In this webinar, you will learn

Webinar Details

  • Title: Session 1: SLEEK: A highly efficient transgene knock-in technology in clinically relevant cell types
  • Speaker: Dr. John Zuris, Director of Editing Technologies – Editas Medicine
  • Date: September, 20th 2023
  • Time: 11:00AM EDT / 8:00AM PDT
  • Inefficient knock-in of transgene cargos limits the potential of cell-based medicines. This presentation describes a study in which a CRISPR nuclease that targets a site within an exon of an essential gene was used to design a cargo template so that correct knock-in would retain essential gene function while also integrating the transgene(s) of interest. This technology, called SLEEK (SeLection by Essential-gene Exon Knock-in), achieved knock-in efficiencies of more than 90% in clinically relevant cell types without impacting long-term viability or expansion
    • Free Registration
  • Title: Session 2: Characterization and biodistribution of REGENXBIO NAV® platform capsids: Under-employed gene therapy vector AAV7
  • Speaker: Dr. Samantha Yost, Senior Scientist - REGENXBIO
  • Date: October 4th, 2023
  • Time: 11:00AM EDT / 8:00AM PDT
  • The use of adeno-associated viruses (AAV) as gene delivery vectors has vast potential for the treatment of many severe human diseases. A small group of intensively studied AAV capsids have been propelled into pre-clinical and clinical use, and more recently, marketed products; however, many less-studied, naturally occurring capsids may also have desirable properties that have yet to be thoroughly described. Within this under-studied group of capsids is AAV7, a potentially therapeutic capsid for which tropism studies are limited, and direct, head-to-head comparisons with well-described capsids have not been performed. This presentation describes a study to characterize the biodistribution of this capsid more fully by several methods including quantitative PCR, immunofluorescence (IF) and immunohistochemistry (IHC), and whole-body imaging via cryofluorescence tomography (CFT) as an unbiased method to look for novel regions of transduction.
    • Free Registration
  • Title: Session 3: Harnessing iNKT cells for ‘off-the-shelf’ cancer therapy
  • Speaker: Dr. Lil Yang, Associate Professor - University of California
  • Date: October 18th, 2023
  • Time: 11:00AM EDT / 8:00AM PDT
  • Invariant natural killer T (iNKT) cells are ideal cell carriers for developing allogeneic cell therapy because they are powerful immune cells targeting cancers without graft-versus-host disease (GvHD) risk. However, healthy donor blood contains extremely low numbers of endogenous iNKT cells. This presentation will describe a study combining hematopoietic stem cell (HSC) gene engineering and in vitro differentiation, to generate human allogeneic HSC-engineered iNKT (AlloHSC-iNKT) cells at high yield and purity. These cells closely resemble endogenous iNKT cells, effectively target tumor cells using multiple mechanisms, and exhibit high safety and low immunogenicity. Collectively, these preclinical studies demonstrate the feasibility and cancer therapy potential of AlloHSC-iNKT cell products and lay a foundation for their translational and clinical development.
    • Free Registration

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