Original Agenda
We are actively working with our speakers to confirm their availability for the virtual event. Initial response from our speakers has been very positive, and we are optimistic we will have the new programs ready to share here soon.

Cell and Gene Therapy Analytics


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 process technologies are required to support preclinical and clinical development, quality control strategies and progression through the regulatory steps needed to reach the market. The PEGS Cell and Gene Therapy Analytics conference offers an all case study format in which leading scientists will share their experiences with new tools, lessons and learnings – and what works and doesn’t work to move efficiently in this explosive new field of pharmaceutical science.

Final Agenda

THURSDAY, september 3

12:00 pm Registration

12:35 Luncheon in the Exhibit Hall with Poster Viewing

CHARACTERIZATION ASSAYS

1:40 Chairperson’s Opening Remarks

Lake Paul, PhD, President, BioAnalysis, LLC

1:50 Assay Development for Emerging Single-Cell Platforms

Eric Alonzo, PhD, Senior Scientist, bluebird bio

Clinical-grade CAR T cell drug products contain a heterogenous mixture of phenotypically and functionally distinct cells. Such heterogeneity necessitates innovative and comprehensive strategies to characterize CAR T cell therapy investigational drug products. Here, we present how high-dimensional single-cell analytics in CAR T manufacturing and beyond can be used to resolve drug product complexity and identify potentially key clinical correlates.

2:20 Characterization of Gene Therapies by Charge Detection Mass Spectrometry

Martin Jarrold, PhD, Professor, Chemistry, Indiana University

Conventional mass spectrometry is usually limited to masses less than a Megadalton because of heterogeneity. Charge detection mass spectrometry (CDMS) can overcome this limitation and extend accurate mass measurements into the Gigadalton regime. Recent technical advances have dramatically improved the sensitivity of CDMS. Applications of CDMS to the analysis of complex biopharmaceuticals, including gene therapy products and vaccines, will be presented.

2:50 KEYNOTE PRESENTATION: T Cell-Intrinsic and -Extrinsic Mechanisms Response to CAR T Cell Therapy

Jan Joseph (Jos) Melenhorst, PhD, Adjunct Associate Professor, Pathology & Laboratory Medicine, University of Pennsylvania

This past decade has witnessed the transformation of cancer therapy via chimeric antigen receptor (CAR)-redirected lymphocytes. Translational sciences have shed light on the mechanistic basis of response and resistance, which are translated into the next-generation CAR therapies. I will summarize some of these principles and how those inform next-generation CAR T cell therapies for liquid and solid tumors.

Charles-River 3:20 A Platform Approach for Analytical Methods to Support Adeno-Associated Virus (AAV) Gene Therapy Products

Yan Zhi, Director Scientific Services, Biologics, Charles River

Recombinant adeno-associated virus (rAAV) vectors have been widely used for in vivo gene therapy with commercial products approved by FDA. Charles River Laboratories (CRL) PA Biologics has established a platform approach for rAAV vector genome titer quantification, residual host cell DNA quantification and sizing evaluation, and replication competent virus (rcAAV) detection, by adopting real-time PCR as well as ddPCR technologies and using reference materials available from ATCC or biological materials created at CRL.

3:50 Networking Refreshment Break

INCREASING ANALYTICAL DEPTH AND THROUGHPUT

4:20 Overcoming the Barriers of Biophysics in Gene Therapy

Lake Paul, PhD, President, BioAnalysis, LLC

Currently the gene therapy space is under utilizing the power of biophysics, specifically Analytical Ultracentrifugation (AUC). Besides the quantitation of empty capsids, AUC can also be used to evaluate the physicochemical properties, higher-order structures, and other pertinent properties. The EMA and FDA are seeing QC release specifications based on AUC, therefore it is paramount that AUC methods are fully realized and meticulously developed.

4:50 Understanding the Effect of Post-Translational Modifications of AAV Capsid Proteins and Their Impact on AAV Infectivity

Lin Liu, PhD, Principal Scientist, Biologics Development, Sanofi

Viral capsid proteins play an important role in cellular targeting and trafficking as part of the viral infection cycle, and thus changes in the viral capsid protein sequence or post-translational modifications (PTMs) might impact viral targeting and infectivity. We evaluated the role of AAV capsid protein PTMs on AAV transduction potential by generating AAV2 and AAV5 capsid mutants and performing a stress study.

5:20 End of Day

5:15 Registration for Dinner Short Courses


5:45-8:15 pm Recommended Dinner Short Course*

SC15: Introduction to Gene Therapy Products Manufacturing and Analytics - Detailed Agenda

*Separate registration required.

FRIDAY, september 4

8:00 am Registration and Morning Coffee

STANDARDS AND REGULATORY CONSIDERATIONS

8:30 Chairperson’s Remarks

Eric Alonzo, PhD, Senior Scientist, bluebird bio

8:35 Principles and Practices for Bioassay Standards

Timothy Schofield, Owner and Consultant, CMC Sciences, LLC

Standards are essential to the development and control of biological products. Considering their importance, there is no consensus on the source of a standard, the basis and means of standard qualification, and stability evaluation. This talk will discuss principles and practices related to standards used to report potency of biological products and propose strategies. Those proposals will borrow from practices related to quality by design, highlighting fitness-for-use of a standard.

9:05 Current Efforts in Bioassay Standardization

Dawn Henke, PhD, Senior Technical Program Manager, Standards Coordinating Body

This discussion will cover standards for regenerative medicine products. Discussion will focus on standards for bioassays. Published standards and how to implement these standards be addressed. An overview of current standards under development and how to get involved, as well as a forum for questions will be provided.

9:35 PANEL DISCUSSION: Standards and Regulatory

Little_ThomasModerator: Thomas Little, PhD, President and CEO, Bioassay Sciences, Thomas A. Little Consulting


walfish_StevenPanelists: Steven Walfish, MBA, Principal Scientific Liaison, USP


schofield_timothyTimothy Schofield, Owner and Consultant, CMC Sciences, LLC


Henke_DawnDawn Henke, PhD, Senior Technical Program Manager, Standards Coordinating Body


  • Setting standards for new bioassay modalities including cell & gene therapies and immunotherapies
  • Developing standards and using already-established standards
  • Getting involved with standard-setting organizations
  • Qualification of standards
  • Correction of relative potency when changing standards

10:05 Networking Coffee Break

PRODUCT QUALITY ATTRIBUTES

10:35 Analytical Methods for Quality Assessment of Cell-Based Medicinal Products

Wim Jiskoot, PhD, Professor, Drug Delivery Technology, Leiden University, The Netherlands

My presentation will start with a brief introduction into quality attributes of cell-based medicinal products, including an overview of routine (QC) methods used to characterize such products. Next, I will discuss the potential applicability of image-based techniques to assess the viability and purity of cells, which I will illustrate with results obtained in my lab.

11:05 Challenges and Hope for the Chemical and Biological Characterization of AAV-Based Gene Therapy Products

Dong Xu, PhD, Senior Scientist, Biogen

Two case studies are included in this presentation to assess Critical Quality Attributes (CQA) for AAV-based gene therapy products; one focuses on the physical and chemical aspects for the capsid vector, while the other quantitatively measures the intracellular activity of target gene. They represent the current methods of choice and are applicable to platform analytical assay development for similar therapeutic programs.

POTENCY ASSAYS

11:35 Overcoming the Limits and Variability of Cell and Gene Therapy Potency Assays

Cheung_WinWin Cheung, PhD, Associate Director, Analytical Development, REGENXBIO

The development and validation of robust potency assays continues to be a significant challenge in the preclinical development of cell and gene therapies. This presentation outlines potential strategies for overcoming the limit and variability issues for these important studies.

12:05 pm High-Throughput Potency Methods Applied to Formulation Development of Live Viral Vaccines

Prashant Kumar, PhD, Senior Scientist, Vaccine Analytics, University of Kansas

We evaluated quantitative Reverse-Transcription Polymerase Chain Reaction (RT-qPCR) as an alternative to traditional Fluorescent Focus Assay (FFA) for measuring live virus in vitro potency. A high correlation between the two assays, no interference of pharmaceutical excipients, and the advantages of RT-qPCR assay (improved speed, throughput, and ease), together demonstrate the advantages of the RT-qPCR assay over FFA to more rapidly monitor and compare live virus vaccine stability in different formulations.

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:05 Networking Refreshment Break

ANALYTICAL CHALLENGES IN ADVANCING CELL AND GENE THERAPIES

1:35 Chairperson’s Remarks

Dong Xu, PhD, Senior Scientist, Biogen

1:40 Evaluating a Control Strategy for an Autologous Cell Therapy for Risk to Patients

Ken Riker, Director, Product Quality, Celgene

An integrated control strategy was developed for a late-phase CAR T cell therapy product based on our current understanding of the product quality attributes, the manufacturing process, and analytical capability. Based on this comprehensive assessment, the overall risk of drug product to patient safety and product efficacy was determined to be low, demonstrating that the combined operational and testing controls are suitable to ensure product quality from the proposed commercial process.

2:10 Machine Learning for the Rapid Classification of Flow Imaging to Characterize Cell Therapies

Amber Fradkin, PhD, Director, Particle Characterization Core Facility, KBI Biopharma

We used a very common analytical method to characterize subvisible particles in biologics (Micro-Flow Imaging) and applied the technique to cell therapy products. We have developed customized machine learning algorithms specific to cell therapy MFI data for rapid classification of images to establish particle profiles. The method has overwhelming potential to monitor cell particle size distributions, cell debris, cell agglomerates, as well as extrinsic material from batch to batch.

2:40 Deep Analysis to Support Early Stage Product and Process Characterization and Improved Release Assays

Alden Ladd, Scientist, Cellular Process Development, bluebird bio

Typical release assays for CAR T products have been shown to not be predictive of clinical outcomes. Characterization activities should integrate single-cell analytics and sensitive functional assays to define surrogate biomarkers of potency that can be used in release assays, process understanding, process improvement, and patient selection. As product complexity increases, new surrogate potency assays will need to be developed to address the multifaceted modes of action of these complex products.

3:10 Development and Qualification of a Cell Counting Assay for Dose Determination

Michele Perry, Lead Microbiology Associate, Analytical Development, Synlogic

Live bacterial products (LBP) are novel therapeutics with the potential to treat unmet needs in rare genetic diseases, such as phenylketonuria. Viability is a critical aspect of LBPs and traditionally determined through colony-forming unit methods. Another approach to determining viability is automated cell counting. When the methods were compared, the cell counting results were more indicative of in vivo activity of the LBPs and is now used to determine dose.

3:40 End of Conference


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