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

Thursday, September 3

CHARACTERIZATION ASSAYS

12:45 pm

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.

1:05 pm

Assay Development for Emerging Single-Cell Platforms

Eric S. Alonzo, PhD, Senior Scientist, Cell Analytics, 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.

Michael Hantman, Associate Director, Methods Development, 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.

1:50 pm LIVE Q&A:

Session Wrap-Up

Panel Moderator:
Eric S. Alonzo, PhD, Senior Scientist, Cell Analytics, bluebird bio
Panelists:
Martin Jarrold, PhD, Professor, Chemistry, Indiana University
Michael Hantman, Associate Director, Methods Development, Biologics, Charles River
2:10 pm Refresh Break - View our Virtual Exhibit Hall

STANDARDS AND REGULATORY CONSIDERATIONS

2:25 pm

Chairperson's Remarks

Steven Walfish, Principal Scientific Liaison, Global Science & Standards, USP
2:30 pm

Principles and Practices for Bioassay Standards

Tim Schofield, Owner & 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.

2:50 pm

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.

3:10 pm PANEL DISCUSSION :

Standards and Regulatory Considerations

Panel Moderator:
Steven Walfish, Principal Scientific Liaison, Global Science & Standards, USP
Panelists:
Dawn Henke, PhD, Senior Technical Program Manager, Standards Coordinating Body
Tim Schofield, Owner & Consultant, CMC Sciences LLC
3:50 pm Close of Day

Friday, September 4

INCREASING ANALYTICAL DEPTH AND THROUGHPUT

9:05 am

Overcoming the Barriers of Biophysics in Gene Therapy

Lake Paul, 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.

9:25 am

Understanding Structure-Function Relationship on AAV Capsid Proteins in Gene Therapy Products

Lin Liu, PhD, CMC Dossier Development & Coordination, 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.

Kevin Lance, PhD, Marketing Manager, Marketing, Unchained Labs

When thermally stressed AAV genomes leak out from their capsids, and do it before proteins melt. However, methods that look only at protein unfolding don’t track DNA, so you’re not getting the whole story. This talk will show Uncle’s insight on AAV stability by monitoring DNA ejection from capsids, and how to look at quantifying encapsulated DNA and particle titer.

10:10 am LIVE Q&A:

Session Wrap-Up

Panel Moderator:
Lake Paul, President, BioAnalysis LLC
Panelists:
Lin Liu, PhD, CMC Dossier Development & Coordination, Sanofi
Kevin Lance, PhD, Marketing Manager, Marketing, Unchained Labs
10:30 am Speed Networking Coffee Break - View our Virtual Exhibit Hall

ANALYTICAL CHALLENGES

10:50 am

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

Ken Riker, Director Product Quality, Product Quality, Celgene Corp.

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.

11:10 am

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

Amber H. 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.

11:30 am

Development and Qualification of a Cell Counting Assay for Dose Determination

Michele Perry, Lead Microbiology Associate, Analytical Development, Synlogic, Inc.

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.

11:50 am LIVE Q&A:

Session Wrap-Up

Panel Moderator:
Ken Riker, Director Product Quality, Product Quality, Celgene Corp.
Panelists:
Amber H. Fradkin, PhD, Director, Particle Characterization Core Facility, KBI Biopharma
Michele Perry, Lead Microbiology Associate, Analytical Development, Synlogic, Inc.
12:10 pm Close of Summit





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