June 29th, 2021 11:00 AM EDT

Generating CAR or TCR T Cells to clinical scale in a quick GMP-compatible manufacturing workflow using nS/MARt DNA Vectors with MaxCyte Flow Electroporation

Matthias Bozza

The compelling need to provide adoptive cell therapy (ACT) to an increasing number of oncology patients within a meaningful therapeutic window makes the development of an efficient, fast, versatile, and safe genetic tool for creating recombinant T cells indispensable. Despite the extraordinary efficacy shown by recombinant T cells expressing Chimeric Antigen Receptors (CARs) in numerous clinical trials, significant challenges remain, ranging from a long lead time, and expensive manufacturing to complicated vector-engineering, optimized gene expression and delivery, and reduced vector-mediated toxicities The DNA Vector Lab at the DKFZ has developed Nano-S/MARt (nS/MARt), a novel DNA Vector platform that combines prolonged CAR-TCR expression with minimal disruption of T cell activity. This antibiotic-free, nanovector technology uses scaffold/matrix attachment regions (S/MARs) for DNA vector maintenance and replication and it can be introduced efficiently into primary human T Cells without toxicity. When combined with GMP-compliant MaxCyte Flow Electroporation® and CliniMACS Prodigy™ automated cell processing, the nS/MARt platform enables the production of clinically relevant recombinant CAR or TCR T cells which provide enhanced anti-tumor activity in a single week.

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Apr 7, 2021 09:30 AM EDT

Development of a genome-wide CRISPR screen in CD4+ T cells to identify drug targets for immune-mediated inflammatory diseases

Ricky Trigg
 Dr. Trigg is a Senior Scientist in the Department of Functional Genomics at GSK.

Immune-mediated inflammatory diseases (IMIDs) are a group of disorders characterized by tissue inflammation as a result of dysregulated immune responses. This webinar will showcase the development of a genome-wide CRISPR screen in primary T cells to identify drug targets for IMIDs, and will cover key areas of workflow optimization, including lentiviral transduction, Cas9 electroporation in bulk cell populations, and fluorescence-activated cell sorting. Preliminary data obtained from a recent ‘mini screen’ will be discussed and the session will close with some key learnings from the workflow development process.

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October 15th, 2020 11:00 AM EDT

Correction of the Sickle Cell Disease Mutation with CRISPR/Cas9

Dr. Mark DeWitt
Project Scientist at the University of California, Los Angeles

Sickle Cell Disease (SCD), one of the world’s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. A universal curative therapy for SCD would address a critical unmet medical need in the United States and worldwide. During this webinar, Dr. Mark DeWitt will discuss the development of a CRISPR/Cas9-based strategy to correct the mutation in CD34+ HSPCs harvested from SCD patients. This technique does not rely on viral vectors that can be challenging to manufacture, instead using synthetic reagents: a Cas9 ribonucleoprotein (RNP) targeting the sickle mutation, and a short single-stranded DNA to program gene correction via homology-directed repair (HDR). These studies will be used to support a 2020 IND filing to initiate a Phase I clinical study in 2021, the first of its kind using CRISPR/Cas9-mediated homology-directed repair in hematopoietic stem/progenitor cells. He will conclude with a critical assessment of the current state of sickle cell disease gene therapy, including progress we have made and challenges that still remain.

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September 17, 2020 8:00 AM PT

Scalable Manufacturing and Nanovesicle Delivery of CRISPR-Cas9 Ribonucleoproteins Using a cGMP- Compliant Cell Engineering Platform

Peter Gee
MaxCyte Field Application Scientist

CRISPR-Cas9 has tremendous potential as a therapeutic tool for treating human diseases. However, prolonged expression of the nuclease and gRNA from viral vectors in an in vivo context may cause unwanted off-target activity and immunogenicity. To overcome these safety issues, a system was recently developed for transient delivery of CRISPR-Cas9 ribonucleoprotein (RNP), recruiting Cas9 protein by chemically-induced dimerization and sgRNA via a viral RNA packaging signal into extracellular nanovesicles.  This system, termed NanoMEDIC (nanomembrane-derived extracellular vesicles for the delivery of macromolecular cargo), demonstrates efficient genome editing in various hard-to-transfect cell types, including human induced pluripotent stem (iPS) cells and myoblasts, and also in vivo in a luciferase reporter mouse model.

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August 04, 2020 11:00 AM EDT

Flexible, clinically adaptable, non-viral approaches to CAR TCR methodologies

Rama S Shivakumar
Research Scientist and Customer Application Specialist, MaxCyte Inc.

In this webinar for scientists and researchers, Rama Shivakumar, a senior scientist at MaxCyte Inc., will highlight powerful case studies that demonstrate the successful use of MaxCyte’s clinically validated, scalable electroporation system in the pre-clinical and clinical scale engineering of resting and activated T cells using a mesothelin specific CARmRNA; in the enhancement of NK cell cytotoxicities against B cell malignancies using an antiCD19 CAR mRNA; in the transposon (Piggybac and Sleeping Beauty) based gene delivery for manufacture of CAR-T cells ; and finally in the gene editing of T cells for improving the efficacy of a TCR immunotherapy. In particular, during this webinar she will discuss how MaxCyte’s versatile ExPERT platform can enable the next-generation non-viral CAR T therapies including allogeneic, off-the-shelf modalities with the potential for enhanced effectiveness for refractory cancer.

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