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Innovations in cell and gene therapy development and the technologies that have advanced the field

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This special feature highlights interviews and resources about the latest advances in cell and gene therapy and the technologies behind them

9 Oct 2023
Lawrence Howes
Editorial Assistant

Editorial article

Microbiome Special Feature

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.

1. Services to support your gene and cell 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.


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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.


Read article


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.


Download method


4. Increase productivity in cell and gene therapy bioprocessing

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.


Download whitepaper


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.


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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.


Download eBook

Graphic of DNA to represent the methods used in cell and gene therapy
Cell and gene therapy is an exciting area of biopharma that offers the potential to treat, prevent, or cure diseases with underlying genetic causes. © Sergey Nivens @123rf.com

Further resources to enhance cell and gene therapy discovery and development

  • Bioprocessing : Learn how you can harness the latest bioprocessing technology in cell and gene therapy in this interview with Lina Tao, Vice president, Eppendorf SE Bioprocess Unit, and learn how 3D-stirred bioreactors could become the industry’s new gold standard. Read article >>
  • Quality control: In this guest editorial, BioPhorum explores potential improvements to ensure quality in cell, gene and RNA therapy development and to overcome the issues associated with guidance and standardization that are still being developed. Read article >>
  • Cancer treatment: In this Q&A, Jai Balkissoon, M.D., FACS, Head of Cell and Gene Therapy, Thermo Fisher Scientific, and Dr. Evan Zynda, Sr. Staff Scientist, Thermo Fisher Scientific, highlight the key trends and challenges in cell and gene therapy. Plus, they explore the latest advancements in cell therapy for cancer treatment. Read article >>
  • Virtual summit: Access the SelectScience Biopharma Summit on demand to watch presentations on topics including the standardization of traceability of personalized cell and gene therapies, innovation and collaboration in regulatory science, and more. Watch on demand >>
  • AI & mass spec: Dr. Mimi Roy, a leading scientist working at the cutting edge of developing gene therapies for rare diseases, discusses the basics of gene therapies and viral vectors and why AI and mass spectrometry are advancing the field in this SelectScience Accelerating Science podcast. Listen to podcast >>

Read reviews on all the products advancing cell and gene therapy research

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."

Photo of the NucleoCounter® NC-200™ automated cell counter from ChemoMetec
The NucleoCounter® NC-200™ automated cell counter from ChemoMetec


Ease of use: 5/5

After sales service: 5/5

Value for money: 5/5





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NucleoCounter® NC-200™ automated cell counter

ChemoMetec

The NucleoCounter® NC-200™ is a high precision automated cell counter utilizing state-of-the-art image cytometry. With user-adaptable protocols and specialized assays for counting a wide range of mammalian cells, including adipose-derived stem cells, T cells, aggregated cells and cells on microcarriers, the NucleoCounter® NC-200™ provides an all-in-one solution for cell counting and cell viability determinations.

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Genome AnalysisGenomics, the study of genomes, includes functional genomics, evolutionary genomics and comparative genomics. There are many genomic technologies such as DNA sequencing of whole genomes, computational biology and bioinformatics. DNA and nucleic acids must be isolated and concentrated from cells for analysis with kits, automated analyzers and software. Other useful technologies for studying genomics include PCR, microarrays and electrophoresis.Gene Expression and Molecular CloningMolecular cloning is a set of techniques that utilizes vectors to transfer recombinant DNA into host cells and is an essential tool for investigating the expression of genes and proteins in bacterial or mammalian cells. A variety of vectors optimized for gene cloning and expression in a range of host organisms are available, alongside competent cells for genetic replication. Here, you can explore a range of molecular tools, high-quality genomic and cDNA libraries, premade clones, transformation and transfection reagents and mutagenesis or gene expression detection assays and expression arrays. Find the best gene expression and molecular cloning products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Next Generation SequencingNext-generation sequencing (NGS), also known as whole-genome sequencing, high-throughput sequencing and massive parallel sequencing, produces and analyses thousands to millions of nucleotide sequences at once. Sequencing systems operate via varying technologies depending on the manufacturer, including sequencing by synthesis, ligation, pyrosequencing, ion semiconductor and single-molecule real-time sequencing. For NGS, library preparation is paramount to successful sequencing. In this section, explore a range of library preparation kits, from targeted, amplicon-based or hybridization-based kits including epigenomic, transcriptomic and genomic workflows to fragmentation kits. Find the best next-generation sequencing products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Protein ExpressionProtein expression is the utilization of cell machinery for the synthesis of proteins and has become a critical tool in biotherapeutic, genomic, and proteomic research. Produce recombinant proteins with expression vectors in combination with a host cell suitable for high-level protein expression. For production of toxic proteins, consider cell-free expression vectors. Create and monitor post-translational modifications with protein modification kits. Find the best protein expression products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Clinical GeneticsMolecular Genetics covers the analysis of hereditary genetic disease and chromosomal abnormalities. Genetics can be analysed using DNA, RNA, and protein microarrays, PCR, RT PCR and DNA sequencing. Genetic equipment includes genetic workstations, thermal cyclers, cooling blocks and electrophoresis products. Diagnostic kits are used for DNA / RNA extraction and purification.Cell TherapyCell therapy involves using living cells to treat diseases, often by replacing damaged cells or stimulating regeneration. Stem cell therapy and CAR-T cell therapy are examples of cutting-edge treatments in regenerative medicine and cancer immunotherapy. Browse our peer-reviewed product directory to find the best cell therapy tools, compare products, check reviews, and get pricing directly from manufacturers.Genome EditingGenome editing involves altering the DNA of an organism, which holds promise for disease treatment, agriculture, and gene therapy. Techniques like CRISPR-Cas9 are revolutionizing this field, enabling precise genetic modifications. Explore genome editing tools in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.Gene TherapyGene therapy is a technique that modifies or replaces genes within an individual's cells to treat or prevent diseases. This approach holds potential for curing genetic disorders, cancers, and certain viral infections. Advances in gene editing technologies like CRISPR are driving the growth of gene therapy. Explore gene therapy tools and technologies in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.

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