The State of CRISPR and Gene Editing Presentation

Translating CRISPR-based Therapies to the Clinic with GMP-compliant, Scalable Electroporation

Virtual breakout session
June 5, 2024
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Jim Brady, Senior Vice President of Technical Applications at MaxCyte, presents on how electroporation is an effective tool in engineering scalable cell therapies.

In this enlightening breakout session from The State of CRISPR and Gene Editing summit hosted by Genetic Engineering and Biotechnology News, James Brady, PhD, presents Translating CRISPR-based Therapies to the Clinic with GMP-compliant, Scalable Electroporation.

MaxCyte’s electroporation platform supports cell therapy developers from concept to commercialization. Our Flow Electroporation platform has been used in a wide array of applications and cell types, including CRISPR gene editing, and was developed specifically for use in the clinic.

Key topics covered in this session

  • CRISPR Gene Editing: Understand the power of CRISPR technology in precisely modifying genes for therapeutic purposes.
  • Clinical Case Studies: Explore real-world examples where MaxCyte’s technology played a pivotal role in advancing cellular therapies.
  • Key Advantages: Discover how MaxCyte’s proprietary Flow Electroporation technology enables efficient and scalable delivery of CRISPR components into cells. Learn about the scalability, standardization, and clinical relevance of MaxCyte’s electroporation approach.

Whether you’re a researcher, clinician, or therapy developer, this session provides valuable insights into the future of CRISPR therapies. Let’s dive in!

Watch the The State of CRISPR and Gene Editing breakout session

Cover slide of Translating CRISPR-based Therapies to the Clinic with GMP-compliant, Scalable Electroporation session

Case studies presented

Multiplex CRISPR editing for cancer immunotherapy: During this phase 1 clinical trial, researchers focused on engineering patients’ T cells using CRISPR gene editing to enhance the potency of an autologous anticancer T-cell therapy. MaxCyte’s electroporation platform played a crucial role by effectively delivering CRISPR components into patient-derived T cells to edit three loci – TRAC, TRBC and PDCD1 – followed by viral delivery of a tumor-targeting T-cell receptor. The corrected cells were successfully infused into patients with safe and effective results, which demonstrates multiplex human genome engineering is safe and feasible using CRISPR-Cas9.

Non-viral manufacture of a CAR T-cell therapy for B-cell non-Hodgkin lymphoma using CRISPR knock-in: In this case study, researchers simultaneously knocked out the PD-1 checkpoint inhibitor via CRISPR while introducing a CAR gene into the same locus via homology-dependent recombination. These CAR T-cells were infused into patients with the results of significant tumor regression and improved survival rates.

Autologous cell therapy for sickle cell disease and beta-thalassemia: The BCL11A locus was knocked out using CRISPR to induce adult expression of fetal hemoglobin. MaxCyte’s electroporation platform played a crucial role by efficiently delivering CRISPR components into patient-derived hematopoietic stem and progenitor cells (HSPCs). The corrected HSPCs were successfully transplanted into patients, resulting in improved hemoglobin levels and reduced disease symptoms.

Presenter

James-Brady

James Brady, PhD

Senior Vice President of Technical Applications at MaxCyte

Dr. Brady is an experienced biotechnology industry professional with expertise in gene and cellular therapy, biologics, and drug discovery. Previously, he was a Senior Scientist at Genetic Therapy, a Novartis subsidiary, where he worked on lentiviral-based gene therapy treatments and Group Leader at MetaMorphix, managing the company’s transgenic and genetic research programs.

Dr. Brady earned a Master of Business Administration in finance from The Johns Hopkins University. He completed his postdoctoral fellow at the National Eye Institute of the National Institutes of Health in Bethesda, Maryland, after obtaining a PhD in genetics from Indiana University in Bloomington, Indiana, and a Bachelor of Science in biology from the College of William and Mary in Virginia.

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