Cell and Gene Therapy Analytics track banner

A vast number of mainstream and emerging biotechs are now engaged in the discovery and development of cell and gene therapies, but significant advances in analytical, bioanalytical and process technologies are required to support preclinical and clinical development, quality control and CMC strategies needed to develop these novel modalities. The inaugural Cell & Gene Therapy Analytics at PEGS Europe will look at the strategies, methods and best practices for analytical characterization and assay development of AAVs, lentiviral vectors, allogeneic and allosteric cell therapies.

Pre-Conference Virtual Short Course
14:00 Recommended Pre-Conference Virtual Short Course*
SC2: The Tumour Microenvironment and Response to Cancer Immunotherapy

*Separate registration required. See conference website short course page for details.

Tuesday, 2 November

07:00 Registration and Morning Coffee



Chairperson's Opening Remarks

Ian Anderson, Associate Director, Analytical Sciences, Pharmaron

Development of an Anion Exchange Chromatography Assay for Determining Empty and Full AAV Capsid Content in AAV6.2

Thomas Linke, Senior Scientist, Process Development, AstraZeneca

Non-genome-containing empty AAV capsids are by-products during AAV production that have been reported to potentially impact AAV product safety and efficacy. We present the development of a QC-friendly anion exchange chromatography (AEX) assay for the determination of empty and full AAV6.2 capsid percentages, using the CIMac™ AAV Analytical Column that can detect as low as 2.9% empty capsids in AAV6.2 samples. Additionally, the method is easy to deploy, can be automated, and has been successfully implemented to support testing of various in-process and release samples. This research was developed with funding from the Defense Advanced Research Projects Agency (DARPA).

  • NEW TALK - This Presentation Will be Given for the First Time

LC-MS Characterization of Viral Vector-Based Gene Therapy Products

Jill Bradley-Graham, PhD, Scientist, BioAnalytics Characterization, Sanofi Genzyme

Viral-based gene therapy products are complicated molecules, containing nucleic acid, lipid, and protein elements that require extensive characterization to ensure product consistency, efficacy, and safety. Analytical methods play a key role in process development, process control, and characterization of drug substance or products. An LC-MS method was developed to assess viral protein ratio, post-translational modifications, virus- and cell-associated proteins of viral vector products and help establish key structure-function approaches to broadening the understanding of how these viruses interact with cells.


Innovative Analytical Approaches to Characterising AAVs

Ian Anderson, Associate Director, Analytical Sciences, Pharmaron

Given the complexity of Adeno-associated Virus it poses a very big challenge for the analytics. In this presentation I will cover some of the innovative approaches we have taken to overcome these challenges using a variety of techniques including Mass Spectrometry and Analytical Ultra Centrifugation.

10:00 Coffee Break in the Exhibit Hall with Poster Viewing

Analytical Ultracentrifugation: An Auxiliary Tool for Vector Characterization

Robert Pletzenauer, Head of Process Analytics, Gene Therapy, Process Development, Takeda

Although Analytical Ultracentrifugation is certainly not one of the latest developments within our gene therapy analytics portfolio, it is a very valuable tool, especially for vector characterization. In this presentation, I will touch a bit on the history of AUC and the fundamentals for this technique. Then I will give some practical examples of its universal applicability and finally discuss the potential alternatives.

  • ​NEW DATA - This Presentation Contains New Data
  • NEW TALK - This Presentation Will be Given for the First Time

POSTER HIGHLIGHT: The Use of In Vitro Assays to Assess and Measure Gene Therapy Immunogenicity

Sofie Pattijn, Founder & CTO, ImmunXperts, a Q2 Solutions Company

Gene therapy can induce an unwanted immune response that can have an influence on the efficacy and potency of the treatment. Additionally, pre-existing immunity towards AAV and CRISPR can also neutralize the therapeutic effect. In vitro assays such as T cell proliferation assays can be used to assess this unwanted immunogenicity in an early phase. Innate immune assays can be used for the evaluation of potential impurities and innate response inducing contaminants. Sensitive T and B cell Fluorospot assays can be used to monitor patient' specific immunogenicity.

Nelis Denys, Product Manager, Analytics, Unchained Labs

Getting a quick answer on AAV titer, empty/full ratio, and aggregation state is critical to most decisions in the lab. Stunner’s dye-free, label-free, and standard-free AAV Quant delivers all that data using just 2 µL of sample, in less than a minute - way faster than AUC, ELISA and ddPCR. Learn how Stunner’s AAV characterization delivers hassle-free answers.

12:15 Enjoy Lunch on Your Own



Development of a High-Throughput cIEF Method for Quantification of Full and Empty Viral Particles for Cell and Gene Therapies

Jenifer Kaplan, PhD, Principal Scientist I, Novartis Institutes for Biomedical Research

An important PQA of AAVs is the percentage of full particles versus empty particles lacking the genome. A large percentage of empty particles decreases the transduction efficiency, increases the therapeutic dose, and poses a safety risk. Analytical ultracentrifugation is the standard for measuring percent full, but it is time-consuming and requires a large amount of material. To overcome this, we developed a high-throughput cIEF method that can identify DNA-containing peaks for quantifying the percentages of full and empty particles and compared these results to other established techniques.

  • ​NEW TALK - This Presentation Will be Given for the First Time

Analytical Challenges and Strategies for the Development of Allogeneic Cell-Based Therapies

Kelly Kemp, PhD, Senior Director CMC, ViaCyte Inc.

Cell-based products tend to be novel and more complex compared to other biologics, presenting significant analytical challenges. For example, product understanding is essential as therapies progress through clinical trials; however, the development of bioassays to identify CQAs and evaluate potency and comparability can be limiting. Moreover, some cell-based products have a short shelf-life or small-batch sizes that necessitate alternative testing approaches. These and other analytical development considerations will be discussed.


Relevance of MOA-Specific Potency Assays for CGTP Products. Case Study in b-Thalassemia & Sickle Cell Disease

Ilya A. Shestopalov, PhD, Director, Analytical Development, bluebird bio

Quantitative potency assays were developed to demonstrate correction of b-thalassemia and sickle cell disease properties in an in-vitro cell culture system. Potency was found to be specific to the beta-globin lentiviral vector and dependent on transduction efficiency of the autologous gene therapy drug product, demonstrating ability to reject sub-functional drug products. Considerations for assay development, qualification results, and redundancy to transduction efficiency methods will be discussed.

Jordi Rodó, Ph.D, Research Scientist, Svar Life Science

The analytical demands of cell and gene therapies can be extremely challenging throughout the development phases. The use of bioassays facilitates the different developmental phases as they can be used to determine key parameters including: vector potency, product identity or determination/titration of neutralizing antibodies. Here, we present different strategies for the development of reliable bioassays for specific or more global use by showcasing a selection of our engineered iLite® cells.

Natalia Markova, Segment Leader, Pharma., Malvern Panalytical
Important aspects in development of lipid- and virus-based delivery vectors are identification of optimal process and formulation conditions resulting in stable efficacious products. 
This presentation will focus on the use of light scattering detectors and differential scanning calorimetry for rapid and robust characterization of rAAV samples and lipid nanoparticles:
  • physical and thermal stability 
  • size and titer
  • surface characteristics such as charge
  • payload/ transgene load
15:50 Refreshment Break in the Exhibit Hall with Poster Viewing

Bioanalytical and Purification Strategies


Bioanalytical Strategies and Methodologies for Assessing PK and Immunogenicity of Cell/Gene Therapy Products

Tong-Yuan Yang, PhD, Senior Director & Head, PK and Immunogenicity Assay Development, Biologics Development Sciences, Janssen R&D, LLC

Cell/gene therapies have shown to be the most effective treatment for unmet medical needs, such as for hematological malignancies and inherited diseases. Cell/gene therapies are novel modalities which are expected to have complex bioanalytical strategy for assessing pharmacokinetics and Immunogenicity in clinical studies. The challenges lie in the complex critical questions-driven bioanalytical strategy, unconventional methodologies used for bioanalytical methods, and lack of regulatory guidance on “fit for purpose” method validation. This presentation will provide considerations in developing and implementing bioanalytical solutions to enable advancement of cell/gene therapy platforms in clinical studies.  


Downstream Process of Adeno-Associated Viruses (AAV) – Purification, Characterisation and Formulation

Ruth Rieser, PhD Student, Pharmacy, Ludwig Maximilians University

A growing number of clinical trials and marketing authorizations of rAAV-based therapeutics underlines their value in therapy. Therefore, scalable production, purification methods and reliable analytical methods are mandatory. Here, we present novel chromatography-based purification strategies, which are explored as scalable alternatives to established gradient ultracentrifugation-based protocols. Different analytical options for rAAVs will also be presented and compared. Finally, an outlook on stabilization of rAAVs in different formulations will be given.

  • NEW TALK - This Presentation Will be Given for the First Time
Francisco Ylera, PhD, R&D Team Leader, New Technologies, Bio-Rad Laboratories Inc.

Antibodies play a crucial role in the analysis of CAR-T cells. Particularly of interest is the class of anti-idiotypic antibodies that are directed against the CAR since these enable the specific detection of the modified T cells using flow cytometry. Here we present the generation of specific anti-CAR-T cell antibodies and demonstrate how CAR-T analysis benefits from our modular antibody assembly technology using SpyTag-SpyCatcher protein ligation.

18:05 Welcome Reception in the Exhibit Hall with Poster Viewing

Explore new products and services in our Exhibit Hall, engage with poster presenters, schedule 1-on-1 meetings, and build your research community during this open networking period.

19:05 Close of Cell & Gene Therapy Analytics