Next-generation CRISPR mRNA-LNPs and scale up manufacturing for CAR T cells

Live30 webinars are thirty minute presentations designed to update you on the latest innovations, applications and data in a fast yet interactive format.

Engineering T cells to express the chimeric antigen receptor (CAR) redirects these cells to target tumors, making this a promising cell-based cancer therapy. To engineer universal CAR T cells for allogeneic cell therapy, gene editing can be used to remove risks associated with graft-versus-host disease.

Wild-type or high-fidelity Cas9 mRNA and sgRNA can be loaded into lipid nanoparticles (LNPs) to achieve high efficiency knockout of the T cell receptor (TCR) in human primary T cells. Furthermore, next generation sequencing (NGS) and advanced bioinformatics approaches can be used to quantify genome editing at on- and off-target sites to ensure that no unintended double-strand breaks (DSBs) have occurred.

While conventional delivery methods such as viral vectors and electroporation have various published performance shortcomings, lipid nanoparticles (LNPs) can solve many of these concerns to enable RNA delivery into primary T cells with high efficiency whilst maintaining high cell viability.

In this Live30 webinar, Dr. Reka Geczy from Precision NanoSystems and Bernice Thommandru from Integrated DNA Technologies (IDT) discuss the multi-step engineering of human primary T cells, including high-fidelity genomic editing tools, next-generation sequencing to determine on/off target editing, and the manufacturing and scale-up of RNA-LNPs.

Learn more about the following:

• How CRISPR-Cas9 RNA-LNPs can enable high encapsulation efficiency and strong gene knockouts
• How to scale up while maintaining excellent expression and editing with low T cell toxicity
• How to seamlessly scale up cell culture processes from discovery to pre-clinical applications
• How to use NGS and rhAmpSeq™ CRISPR Analysis System to determine on- and off-target genome editing events

Reka Geczy

Scientist II, Product Development, Precision Nanosystems

Reka holds a PhD in Pharmaceutical Sciences from the University of British Columbia with high impact publications at the interface of microfluidics and drug delivery. After her postdoc at the University of Copenhagen, she joined Precision NanoSystems as a Product Development Scientist. Her team is developing potent and efficient lipid nanoparticle compositions for cell and gene therapies.

Bernice Thommandru

Research Scientist III, Integrated DNA Technologies (IDT)

Bernice Thommandru is a Research Scientist in the Molecular Genetics research group at IDT. She received her MS degree in Molecular Physiology and Biophysics from the University of Iowa studying transcriptional regulation of multi-drug resistance in pathogenic fungi of the lung. At IDT, Bernice has focused on optimizing delivery strategies for CRISPR reagents as well as methods for increasing the rate of homology-directed repair.

Watch now

SPEAKERS

Reka Geczy

Scientist II, Product Development, Precision Nanosystems

Bernice Thommandru

Research Scientist III, Integrated DNA Technologies (IDT)

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