From The Editor | November 17, 2022

ICYMI: Cell & Gene Live, Evaluating Translational Research Tools For CGTs


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1


Translational research is critical to move from basic science to clinical application. And so, translational researchers need the infrastructure, training, and technical expertise necessary to develop and manufacture the various components for novel cell and gene therapies. There are various challenges in translational research from navigating regulatory requirements and finding funding to making the leap from research to the clinic and sourcing technologies that suit your project now as well as in the future.

We are well aware of the challenges in translational research for cell and gene therapies, which is why we developed our most recent Cell & Gene Live. In case you missed it, Evaluating Translational Research Tools For CGTs, featured industry experts Gregory Opiteck, Ph.D., VP, Head of Translational Sciences at Allogene Therapeutics and Douglas Sanders, Ph.D., VP, Head of Translational Medicine at AVROBIO as our expert panelists, and the presentation was chock full of details, examples, and data specific to translational research in cell and gene therapy.

During the presentation, we covered a lot of ground. Opiteck and Sanders talked through translational research tools that are needed to bridge the gap from understanding the disease process to identifying therapeutic targets and testing potential therapies in preparation for clinical research in humans. We also covered assay and vendor selection in support of allogenic CAR-T therapies as well as the various modalities of AAV gene therapy.

Regarding biological assay (bioassays) and biomarkers from a gene therapy perspective, Sanders explained the biggest challenges in early therapy development. Opiteck broke down this information regarding cell therapies. We talked through the biomarkers that are the most informative to drug development, and we identified the roadblocks and challenges associated determining success earlier in the drug development process. Both leaders tackled the difficulties in evaluating pre-clinical research as well risk/benefit ratios when it comes to inherent variability of starting materials. Limited lot size and materials for testing, limited stability, and lack of appropriate reference standards can be a challenge, and so, both leaders provided actionable ways to overcome these limitations.

Regarding vendor selection, as it applies to PK/PD (or Pharmacokinetic-Pharmacodynamic) and immunogenicity measurements, in support of allogenic CAR-T therapy, Opiteck talked through best practices as well as expectations for the short term. Opiteck also offered details around the most important functionalities to look for when selecting vendors.

Sanders explained the difference between AAV-mediated gene therapy and ex-vivo lentiviral gene therapy. He talked through which diseases are a better fit for each modality and why.

If you were unable to attend the presentation in real time, don’t worry, because the on-demand version is available for viewing. And while you’re at it, be sure to review our Cell & Gene Live library. Our line-up of Live events touts CGT leaders from academia and industry alike on the major topics and trends you and your colleagues discuss every day. Here are a few past Live events to check out: The Future of CRISPR-Engineered Stem Cell Therapies; Can Innovation Drive Down Cell Therapy Manufacturing Costs?; The Therapeutic Potential Of Exosomes; Kite Pharma's Ultimate Supply Chain Test - A Q&A With Their 4-Exec Panel On Overcoming The Pandemic's Disruption; Advancements In Targeted mRNA Delivery; Scaling Cell Therapies: Auto vs. Allo; and Win The CGT Talent Fight: How To Find And Retain Experts.

We will continue to deliver the timeliest and data-driven content to you in the formats you prefer. Email me directly to inform me of the topics you want to learn more about in webinar format. 2023 is upon us, and we’re building our webinar content calendar as we speak, and I’d love to hear what you want us to cover.