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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 and bioanalytical technologies are required to support R&D, preclinical and clinical development of these novel modalities. The 2nd Annual Cell & Gene Therapy Analytics at PEGS Europe will highlight the analytical development and control strategies for stage-specific development of AAV gene therapy vectors; as well as advanced techniques and approaches for AAV characterization.

Sunday, 13 November

Registration Open12:00

Recommended Short Course*14:00

SC4: Potency Assays and Comparability for Cell & Gene Therapies
*Separate registration required. See short courses page for details.

Monday, 14 November

Registration and Morning Coffee (Garden Room)07:30




Chairperson's Opening Remarks

Borries Demeler, PhD, Professor, Chemistry & Biochemistry, University of Lethbridge


Stage-Specific Analytical Development for AAV Gene Therapy Vectors

Liz Higgins, PhD, Vice President, Head of CMC, NysnoBio

This presentation will focus on strategies for prioritizing early analytical development work. The discussion will include which assays should be put in place first and ways to use template assays and/or first-generation assays (to be upgraded later in development) to ensure assays are available to assess vector quality and potency for pre-IND animal studies and process development activities.


Developing an Analytical Characterization Plan for AAV Gene Therapy Products

Fabien Dorange, PhD, Head, Analytical CMC, SparingVision

Potency is one of the most critical quality attributes for AAV vectors as it is related to the functionality of the product. This presentation will focus on the strategy for the development of the potency assay for SPVN06, a gene-independent treatment for retinitis pigmentosa.


Characterization of AAV Vectors for Preclinical Use


Alignment on upstream and downstream processes and analytical methods from preclinical to GMP manufacturing reduces time to market and de-risks drug development. The use of standardized assays for the characterization of preclinical vectors provides critical insights on potency, identity, and purity, and strengths the value of PoC and IND-enabling studies performed with those vectors.


POSTER HIGHLIGHT: Design of Experiment, a Powerful Tool to Simplify Viral Production Process Development

Anna Doshina, Development Tech Lead, Exothera

Design of Experiment (DoE) is a powerful tool that allows us to study the impact of multiple factors on process performance in fewer experiments. We have studied the manufacturing of adeno-associated viruses (AAV) in scale-down models focusing on optimization of transfection, lysis, endonuclease treatment, and chromatography using DoE. Virus titer and process impurities were evaluated as main outputs. These experiments helped us select process operating ranges and reagents resulting in effective AAV production and purification.

Coffee Break in the Exhibit Hall with Poster Viewing (Verdi and Vivaldi 1&2)10:30


Viral Control Strategy for Gene Therapy Vectors

Christopher Bravery, PhD, Consulting Regulatory Scientist, Advanced Biologicals Ltd.

Viral vectors provide limited or no scope for viral reduction and elimination steps, meaning the control of viral adventitious agents is limited.  In this presentation, the need to consider a wholistic approach to the control of viral agents will be discussed, along with the types of methodology that might be employed, and why.


In-Process Monitoring and Final Product Characterization of Enveloped and Non-Enveloped Viral Particles by Capillary Electrophoresis Method

Rita Fernandes, Research Fellow, iBET Instituto de Biologia Experimental Tecnologica

This paper presents a highly sensitive method, CE-sodium dodecyl sulfate (SDS) combined with a laser-induced fluorescence (LIF) detector, that uses fluorescent labeling to monitor various bioprocess steps and characterize the final product of different viral vectors. Sample purity and capsid protein content of several recombinant Adeno-Associated Viruses produced in different cell systems were analyzed for their viral protein VP1/VP3 ratio. Notably, this method was also successfully implemented for enveloped viruses such as Maraba virus, a chimeric related-virus and lentivirus, showing excellent resolution and sensitivity for measuring purity or vector characterization.

12:15 Aura+: High-Throughput, Low Volume Product Stability, and Purity Analysis for Gene and Cell Therapies

Paul Dyer, PhD, Field Application Scientist, Halo Labs

Aura+ is the latest instrument designed specifically characterize subvisible aggregates for product quality measurements for gene and cell therapies using as little as 5µL sample. Fluorescent Membrane Microscopy FMM is used to identify aggregates revealing exactly what is in your aggregates. Aura+ can the presence of DNA in AAV aggregation to understand the role of leaky capsids in subvisible particle formation or various cellular markers to assess your cell therapies.

Session Break12:45

12:55 Embedding Quality into Cell Line Development via the Application of an Innovative Picodroplet-Based Single Cell Sorter

Jonathan Dempsey, PhD, Managing Director, Pathway Biopharma Consulting

Generation of a highly expressing, regulatory acceptable clonal cell line is the first key activity in the development of a biopharmaceutical.  Cell line development is labour intensive, time consuming, costly, and subject to stringent regulations to ensure patient safety. We will describe the implementation of innovative picodroplet-based technology which provides a visual record of clonality and clone derivation and also allows for transfected pool enrichment resulting in highly expressing clones.  

13:25 Reducing Gene Therapy Development Timelines with Integrated Plasmid DNA and AAV Platform Process

Denis Burton, Ph.D., Director Business Development, Business Development, Catalent Biologics

Plasmid DNA (pDNA) is a critical starting material for advanced therapies. Leveraging CGMP requirements from an early stage, together with the right tools and analytics to provide the highest quality standards for pDNA, will ensure successful downstream applications such as Adeno-Associated Virus (AAV) from early clinical development to the commercial phase. During this talk, we will share the main analytical development challenges linked to CGMP pDNA production and their impact on AAV programs, with a walk-through of the UpTempo Virtuoso AAV platform, a scalable CGMP-ready platform process that significantly reduces the time from gene to clinic.

Session Break13:55



Chairperson's Remarks

Liz Higgins, PhD, Vice President, Head of CMC, NysnoBio


Multiplex Digital PCR: An Analytical Method for Gene Therapy, Delivering a Broad Versatile, and Meaningful Readout

Peter Eisenhut, PhD, Research Investigator CMC, Takeda

Quantification of vector genome titer by digital PCR is a modern analytical method in gene therapy. Using a single target for titer determination can provide a consistent concentration of the product, but it lacks important information such as data on the full-length transgene. Multiplex dPCR was used to quantify DNA for multiple targets of the vector genome. A quadruplex dPCR method was developed for simultaneous quantification of 4 transgene targets.


Addressing Viral Vector Genome Integrity and Capsid Content by Long-Read Sequencing and Multiplex dPCR

David Dobnik, PhD, Senior Research Associate, Biotechnology & Systems Biology, National Institute of Biology

The production of viral vectors for gene therapy is focused on pure, safe, and efficacious products. Absence of impurities and presence of full vector genomes play a crucial role. We have addressed the problem of AAV vector genome integrity by long-read sequencing and an advanced dPCR multiplex approach. Both technologies provide useful information on genome integrity and presence of impurities, but dPCR can go a step further providing a quantitative result.

  • Vector genome integrity towards becoming a critical quality attribute
  • Nanopore long-read sequencing can help in characterization of AAV samples 
  •  Advanced multiplex dPCR approaches can help in quantification of genome integrity?

Analytical Development of a Gene of Interest (GOI) Expression Bioassay for a Gene Therapy Product to Treat Alzheimer's Disease

Savita Nair, PhD, Senior Manager, Bioassay Development, Sangamo Therapeutics

The microtubule-binding protein tau is a key player in Alzheimer’s disease (AD) and frontotemporal dementia. Using Sangamo’s unique zinc finger protein (ZFP) platform it was recently demonstrated that tau down-regulation with AAV ZFP-transcription factors (ZFP-TFs) rescues neuronal damage around amyloid plaques in a mouse model of Alzheimer’s disease. We adopted a matrix approach to determine potency of our AAV ZFP-TF product based on the mechanism of action of our gene therapy product. Design and analytical development of a quantitative PCR-based assay to measure transgene-specific ZFP mRNA for early product characterization and release studies will be presented.

15:50 Fly through AAV Buffer Exchange and Characterization with Unagi and Stunner

Ross Walton, PhD, Senior Application Scientist for Analytics, Unchained Labs

Getting AAV into a new buffer raises a lot of questions about the final concentration, how much buffer was exchanged, and if sample integrity is still looking good. Unagi provides fast, quantitative buffer exchange where you know your AAV sample will never run dry. Before and after a run, Stunner serves up quick and simple answers on AAV titer, empty/full ratio, and aggregation to make sure you’re getting what you want.

Refreshment Break in the Hall with Poster Viewing (Verdi and Vivaldi 1&2)16:20


Analytical Ultracentrifugation as a Multi-Attribute Release and Characterization Method for AAV-Based Products

George Bou-Assaf, PhD, Scientist, Analytical Development – Product & Technology Development, Biogen

Analytical ultracentrifugation is the gold standard method for the characterization of AAV-based products because it has superior resolving power compared to other biophysical and biochemical methods. In addition, the sedimentation velocity AUC method reports on several attributes at once. Here, we discuss recent developments in AUC methodology which allow reduced sample consumption, higher throughput, and transfer of the method to a quality control environment.

  • AUC reports on several attributes at once, therefore it saves time and effort.
  • Recent developments enabled a doubling in throughput.
  • The method is not restricted to a development environment and can be transferred to a QC setting. 

Characterisation of Lipid Nanoparticles by Analytical Ultracentrifugation

Borries Demeler, PhD, Professor, Chemistry & Biochemistry, University of Lethbridge

Distinguishing loaded from empty lipid nanoparticles (LNPs) is challenging, as their overall size and shape may not change proportionally with RNA loading. However, upon loading, their spectral signature and density vary significantly. Using these properties, we developed two analytical ultracentrifuge(AUC) methods that distinguish LNPs. First, density matching AUC, uses varying D2O:H2O ratios to discriminate empty vs loaded LNPs. Second, multi-wavelength AUC, characterizes LNPs based on hydrodynamic and spectral properties.

Welcome Reception in the Exhibit Hall with Poster Viewing (Verdi and Vivaldi 1&2)18:05

Close of Cell & Gene Therapy Analytics Conference19:05