Cambridge Healthtech Institute’s Inaugural

Cell and Gene Therapy Analytics

A Best Practices Exchange for Essential Assays and Product and Process Quality Strategies

May 7-8, 2020

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.

Preliminary Agenda

CHARACTERIZATION ASSAYS

Assay Development for Emerging Single-Cell Platforms

Eric S. Alonzo, PhD, Senior Scientist, bluebird bio

Characterization of Gene Therapies by Charge Detection Mass Spectrometry

Martin Jarrold, PhD, Professor, Chemistry, Indiana University

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

INCREASING ANALYTICAL DEPTH AND THROUGHPUT

Deep Analysis to Support Early Stage Product and Process Characterization

Richard Rustandi, PhD, Research Fellow, Merck

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

POTENCY ASSAYS

Overcoming the Limits and Variability of Cell and Gene Therapy Potency Assays

Zhenhong Li, PhD, Senior Director, Analytics, Analytics, Regenxbio, Inc.

High-Throughput Potency Methods Applied to Formulation Development of Live Viral Vaccines

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

PRODUCT QUALITY ATTRIBUTES

Analytical Methods for Quality Assessment of Cell-Based Medicinal Products

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

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

Dong Xu, PhD, Senior Scientist, Biogen

REGULATORY UPDATES

Regulatory Update for Cell and Gene Therapy

Jean Stanton, Director, CAR-T Site Liaison, Janssen

International Regulatory Update

William C. Motel, PhD, Associate Director, Regulatory Affairs, IQVIA

ANALYTICAL CHALLENGES IN ADVANCING CELL AND GENE THERAPIES

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

Ken Riker, Director, Product Quality, Celgene

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

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

Development and Qualification of a Cell Counting Assay for Dose Determination

Michele Perry, Lead Microbiology Associate, Analytical Development, Synlogic

* The program is subject to change without notice, due to unforeseen reason.