Cambridge Healthtech Institute’s 3rd Annual

Gene Therapy CMC and Analytics

Regulation and Analysis of Vector-Based Gene Therapies

August 14-15, 2019


Gene therapy is an extremely promising technique for the treatment of incurable diseases such as cancer and genetic disorders such as hemophilia. However, the analysis, characterization and delivery of these unique products remain a key issue.

Cambridge Healthtech Institute’s Gene Therapy CMC and Analytics meeting uncovers the practical challenges facing vector-based gene therapy analytics, assay development, quality control, vector development and delivery.

Preliminary Agenda

REGULATION OF CMC OF VECTOR-BASED GENE THERAPIES

USP Standards Development Activities to Support Gene Therapies

Jim Robertson, PhD, Senior Scientific Liaison, USP

Developing CMC Strategies to Advance a Gene Therapy Pipeline

Rajiv Gangurde, PhD, Director, CMC, Voyager Therapeutics

CHARACTERIZATION AND ANALYSIS

Comparative Studies on the Empty/Full AAV Capsid Using Multiple Analytical Methods

Dong Xu, PhD, Senior Scientist, Analytical Development, Biogen, Inc.

Talk Title to be Announced

Anthony Leyme, PhD, Scientist II, Technical Development, Analytical Bioassay Development, Biogen

Plasmid Control Strategy for Triple Transfection AAV Production

Lawrence Thompson, PhD, Principal Scientist, Analytical Research & Development, BioTherapeutics Pharm Sciences, Pfizer

The Application of Process Analytical Technologies for Advanced Viral Manufacture

Damian Marshall, PhD, Head, Analytical Development, Non-Clinical Operations, Cell & Gene Therapy Catapult UK

Bioassay Development for AAV Gene Therapy Products

Aisleen McColl-Carboni, PhD, Senior Scientist, sanofi

Strategies and Advances in AAV Vector Characterization: Product-Related Impurities

Christine Le Bec, Head of CMC Analytical, Technology Development, Genethon

Panel Discussion: Analytical Strategies for Gene Therapies

Xiaohui Lu, PhD, Director, AAV, Production, CRISPR Therapeutics

Bo Yan, PhD, Senior Scientist, Analytical Development, Beam Therapeutics

Wei-Chiang Chen, PhD, Senior Scientist, Analytical, Solid Bioscience

Quality Control of Gene Therapies

Sebastiaan Van Zalen, PhD, Quality Manager, Clinical Vector Core, Children’s Hospital of Philadelphia


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