Robust and cost-effective characterization and manufacturing presents a core challenge in the commercialization of gene and cell therapies with pressure mounting on CMC, analytical and manufacturing teams to keep up with accelerated development times and cost pressures. Gene and Cell Therapy CMC and Manufacturing examines the critical challenges facing the production, characterization and quality control of gene and cell therapies, with dedicated presentations on rapid CMC development, product and process characterization, upstream and downstream bioprocessing and considerations for personalized and large-scale manufacturing.
VP, Pharmaceutical Development
Ultragenyx Pharmaceutical Inc.
Manager of Regulatory Affairs
Senior Director, CMC Regulatory
Genentech, A Member of the Roche Group
[Transformation from R&D to Manufacturing]
Speaker: James Warren
[Virtual Inspections: Navigating the new Paradigm]
Speaker: Monica Commerford
There are clear applications and broad acceptance of augmented reality technologies in training departments but the use of these systems as part of the inspection process has been limited. Virtual inspections have the capability to revolutionize how both client and regulatory inspections are conducted, especially during times of crisis and interruption to business continuity.
However, this technology (e.g. Microsoft HoloLens and Matterport 3D Mapping) is available today and has the capability to be widely used as the standard for inspections of the future. Strategies for implementing on-site virtual inspections will be discussed during this presentation.
[Re-Thinking Comparability Assessments for Individualized Therapeutics]
Speaker: Kathleen Francissen
Cell and gene therapy products (CGTP) include a wide variety of product platforms and involve many new technologies in their development, production, and control. Some of the existing regulatory guidelines pose challenges for advanced therapeutics, and need to be flexible to enable the proper development of innovative therapeutics. This flexibility should not result in leniency or the lowering of product quality standards, but rather in the adoption of meaningful and appropriate approaches and controls. For example, some conceptually different approaches are needed for demonstrating comparability of individualized products when manufacturing processes are updated. Individualized therapeutics are made-to-order for each patient and include multiple product platforms, such as individualized neoantigen-specific therapies (iNeST). Because each batch of an individualized product has unique properties, the standard approach to demonstrating comparability is not possible. Split-stream manufacturing makes it possible to conduct pairwise comparisons of drug substance and/or drug product batches. If the product is well characterized analytically, then many of the usual approaches to demonstrating comparability can be performed meaningfully. However, for modifications in the neoepitope selection process, which involves genomic analysis of patient tumor and blood samples as well as bioinformatic identification and prioritization of candidate neoantigens, trying to conduct a comparability assessment at the product level is not meaningful. Instead, modifications in the neoepitope selection process need to be evaluated using process performance metrics and other comparisons at the process level.