Many of the hurdles to streamlined, cost-effective manufacture of cell and gene therapy products derive from a lack of standardized methodologies and training around CMC programs. To help address this, ARM has published A-Cell, a multistakeholder collaboration to incorporate Quality by Design (QbD) principles into cell-based therapy CMC programs. A-Cell emulates previous QbD efforts that were applied to the manufacturing of monoclonal antibodies (A-Mab), vaccines (A-Vax), and more recently, gene therapies (A-Gene), bringing best practices in CMC to a case study of a CAR-T therapy manufacturing.
In conjunction with the A-Cell document, ARM hosted a nine-part live webinar series highlighting specific A-Cell chapters.
Speaker:
Pinar Cicalese, Ph.D., Director CMC Commercial Strategy, Immatics
This webinar covers the elements of Quality by Design for cell-based therapies, including the QTPP as a product development and life cycle management tool, and risk assessment approaches to identify product CQAs and critical process parameters (CPPs). The speaker explains how developing a good understanding of the inter-relationships between CPPs, CQAs and clinical outcomes is fundamental to the implementation of an effective control strategy, leading to greater assurance of product quality.
Download this webinar’s Q&A transcript here.
Speakers:
Fabio Fachin, Ph.D., Head, Cell Therapy Process Development, Automation and Digital Technologies, Takeda
Bruce Thompson, Ph.D., Vice President and Technical Lead, Resilience
This webinar discusses the manufacturing process overview of a CAR-T cell product. The speakers describe the manufacturing steps and unit operations, as well as process and technological considerations to produce the desired product yield to meet the clinical dose requirements, while maintaining the product quality.
Download this webinar’s Q&A transcript here.
Speaker:
Kelly Zinn, Ph.D., Director, Product Quality Leadership, Atara Biotherapeutics
This webinar discusses the manufacturing process overview of lentiviral vectors (LVs), including the upstream production & downstream purification, and its use as a drug substance in CAR-T cell manufacturing. The speaker describes the Quality by Design (QbD) approach to process understanding and scaling, as well as LV testing and characterization.
Download this webinar’s Q&A transcript here.
Speaker:
Rupa Pike, Ph.D., Senior Director of Technical Affairs, Advanced Therapies, Thermo Fisher Scientific
This webinar covers approaches to analytical assay development, progress of analytical technologies, and challenges related to process and product characterization for cell-based therapies, with focus on a CAR-T example. A Quality by Design (QbD) approach to phase-appropriate, fit-for purpose assay development will also be discussed.
Speakers:
Ramon Montero, Ph.D., Associate Director, R&D, Akron Bio
Ezequiel Zylberberg, Ph.D., Vice President, Product Development and Planning, Akron Bio
This webinar provides an overview of the ancillary materials (AM) landscape in the Cell & Gene Therapy field, including the principles and risk management strategies. The presenters introduce case studies to further strengthen the case for AM standardization through best practices for AM suppliers and users.
Download this webinar’s Q&A transcript here.
Speaker:
Nolan Polson, Ph.D., Vice President, Strategic Product Quality, GSK
This webinar discusses the development of a robust integrated control strategy for a CAR-T cell therapy product in the setting of current regulatory framework and guidelines. The presenter covers various concepts within risk-based approach to control strategy generation, including various process characterization tools and correlative analysis between product CQAs to clinical outcome.
Speakers:
Erin Goodhue, Ph.D., Director, Clinical Research and Development, Terumo Blood and Cell Technologies
Dalip Sethi, Ph.D., Director, Scientific Affairs, Terumo Blood and Cell Technologies
Elizabeth Valdez, Integration Manager, Therapeutic Services, Terumo Blood and Cell Technologies
This webinar covers various aspects of the cell collection process as an integral part of a cell therapy manufacturing process. The speakers provide an overview of the apheresis process, instrument technical operation and optimization techniques, patient preparation and management, donor considerations for allogeneic products, and regulatory considerations from the clinic operations perspective. The webinar concludes with a panel discussion and Q&A session to discuss standardization around apheresis process.
Download this webinar’s Q&A Transcript here.
Speaker:
Peter Walters, Fellow, Advanced Therapies, CRB
This webinar aims to describe the operational aspect of cell therapy manufacturing from the facilities perspective. The speaker provides an overview of the facility design process, design layout, operational design considerations, and qualification, validation, and manufacturing start-up processes to evolve initial facility requirements into a fit-for-purpose operational facility for cell-based therapies.
Download this webinar’s Q&A Transcript here.
Speakers:
Michael Mendicino, Ph.D., President, Chief Consultant & Advisor, Hybrid Concepts International
Emmanuelle Sabbah-Petrover, Ph.D., Director, Regulatory Science, Cell & Gene Therapeutic Products, Voisin Consulting Life Sciences
This webinar covers the pathways for regulatory review and approval for cell and gene therapies in the United States and Europe. The speakers discuss the regulatory frameworks, sponsor interactions with Health Authorities, accelerated regulatory pathways, as well as submission content for clinical trials in these two major markets.
