Cell and gene therapy is an exciting area of biopharma that offers the potential to treat, prevent, or cure diseases for which there is currently no other therapeutic option – for example, rare diseases and cancers. As the field develops, new methods and technologies for ensuring cell and gene therapy safety and efficacy are essential.
Cell therapies involve altering cells outside of the body so that they can restore normal function when introduced back into the patient. Gene therapy, on the other hand, involves changing or restoring the genetic function of a cell by introducing new genetic material, typically delivered by a vector that targets specific patient cells in vivo. By addressing the underlying genetic causes of disease, cell and gene therapies provide targeted treatment.
The analysis of both cell and gene therapy products and their effect requires the application of many advanced techniques and technologies – from plasmid analysis for gene therapy vectors, to the immunoassays used in bioprocess analytics, and monitoring immune response to gene therapies. Together these studies will help to optimize the development of new life-changing treatments as well as the methods that will help bring cell and gene therapy products to market faster.
In this SelectScience® special feature, explore some of the latest technologies and methods used to advance cell and gene therapy development.
From target selection and validation, to CRISPR editing and payload optimization, transgene delivery optimization, and solutions for preclinical, and clinical stages, discover how GenScript’s products and services can support development and manufacturing.
2. The importance of investigating complement system in gene therapy
Dr. Anna Majowicz, Preclinical Immunology Lead, Spark Therapeutics, discusses work to identify and characterize immune responses that might occur after the infusion of AAV-based gene therapies and how this will help to develop safe and efficacious therapies.
3. Plasmid isoform analysis in 5 minutes
Discover a simple and rapid method for discriminating between covalently closed circular, open circular, and linear forms of plasmid DNA using anion exchange chromatography on a TSKgel® DNA-NPR column from Tosoh Bioscience.
In this whitepaper, Gyros Protein Technologies investigates the analytical requirements of viral vector immunoassays used in cell and gene therapy in detail, and explores how automated and reliable immunoassays can lead to increased productivity.
5. Assessment of integrity and purity of mRNAs from lipid nanoparticles
To ensure the quality of LNP-based drugs, comprehensive characterization of these sophisticated molecules is paramount. Explore how to achieve high-quality, robust assessment of the integrity and size of mRNA molecules encapsulated in LNPs with this technical note from SCIEX.
6. Overcome development and manufacturing challenges of T cell-based therapies
In this comprehensive eBook, learn more about the biological and manufacturing challenges for T cell therapies, and discover products, services, and a resource from Bio-Techne that can help overcome these obstacles at each process stage.
Find out what researchers around the world are saying about the products they use, including Andreas Mitsch, of Leibniz-Institut für Immuntherapie, who shared his thoughts on the NucleoCounter® NC-200™ automated cell counter from ChemoMetec and its use in the manufacture and quality control of cell therapy products.
"We replaced our manual cell counting procedure with the NC-200 for in-process and final product controls of ATMP manufacture. High increase in reproducibility and reduction of analysis time greatly supported optimization of cell therapy manufacturing process."
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