2023 PDA Advanced Therapy Medicinal Products Conference

08:00 – 09:45 | P1: Advancing Innovation from Research to the Clinic
Moderator: Friedrich von Wintzingerode, PhD, QC Lead iNeST (individual Neoepitope Specific Therapy), Genentech, Inc.

In this session, we will begin with an overview of the science of cell therapy and how that science is continually evolving in an effort to address challenges across many indications. The session will conclude with a discussion of how clinical and regulatory strategies intersect with CMC  in order to advance these promising therapies toward approval and beyond.

10:30 – 12:00 | P2: Managing Complex Manufacturing Networks for ATMPs
Moderator: Richard Denk, Senior Consulting Aseptic Processing & Containment, SKAN AG

Managing complex cell and gene therapy networks involves coordinating and optimizing a variety of interconnected processes and stakeholders involved in the development, manufacturing, and delivery of cell and gene therapies.

Some key considerations for managing complex cell and gene therapy networks include:

1. Collaborative partnerships: cell and gene therapy networks often involve multiple stakeholders, including academic institutions, research organizations, manufacturers, and regulatory bodies.
2. Regulatory compliance: cell and gene therapies are subject to a variety of regulatory requirements, including those related to safety, efficacy, and manufacturing.
3. Manufacturing process optimization: the manufacturing of cell and gene therapies is a complex process that often involves multiple steps and technologies.

13:15 – 14:45 | P3: Cell Banking and ATMP Development
Moderator: Dayue Chen, PhD, Head of Cell Therapy Technical Development, Genentech, Inc.

As in the case of biologics, establishment of cell banking systems is essential for the development of many ATMP products such as stem cell-based (e.g., iPSC) allogeneic products and viral vector-based gene therapies. It is a critical element of the overall control system that assures consistent product quality and robust process performance. While a master cell bank alone is sufficient in supporting the early phase of clinical development and manufacturing, a two-tiered cell banking system composed of master cell bank (MCB) and working cell bank (WCB) is needed to support commercial manufacturing. However, the topic of cell banking has not received much public attention in the ATMP community. In this session, our presenters will discuss various aspects of cell bank systems in the context of ATMP development and commercialization with the focus on regulatory expectations, MCB/WCB manufacturing, MCB/WCB testing and characterization, and timing of MCB and WCB manufacturing.

15:30 – 17:00 | P4: Supply Chain Considerations Specific for ATMPs
Moderator: Friedrich von Wintzingerode, PhD, QC Lead iNeST (individual Neoepitope Specific Therapy), Genentech, Inc.

ATMPs pose a unique challenge to companies' supply chains. This session will provide insight into ATMP supply chain complexity and challenges to building scale into the supply chain, the importance of ATMP cost-of-goods (COGS), as well as specific examples and case studies related to maintaining quality across multiple outsourced partners and material shortages and long lead times.

07:00 – 08:15 | Breakfast Session: Points to Consider When Delivering ATMPs to Patients
Moderator: Friedrich von Wintzingerode, PhD, QC Lead iNeST (individual Neoepitope Specific Therapy), Genentech, Inc.

This session provides an insight into the challenges companies face when manufacturing ATMPs and delivering them to patients. We will hear how an evolving global regulatory environment is challenging CAR-T manufacturing and items to consider when building a vein-to-vein program for ATMPs under the U.S. FDA’s Expanded Access Program.

08:30 – 10:00 | P5: Industry and Agency Perspectives for Analytical Method Lifecycle Steps
Moderator: Stephan O. Krause, PhD, Executive Director, AS&T, CT Global Quality, Bristol Myers Squibb

This session will cover industry and agency perspectives for suitable analytical method development and lifecycle approaches for accelerated ATMP product development and commercialization processes. Phase-appropriate method development, validation, transfers, and change/improvement strategies will be discussed with regarding to data expectations, and other critical points to consider for successful regulatory submissions. Valuable lessons learned from working in an accelerated development environment with a common goal to bring life-saving therapies to patients will be provided from industry and agency SMEs.

10:45 – 12:15 | P6: Raw Material Management and Control
Moderator: Lori Dingledine, QC Lead- Microbial Control and Compendial Testing, Spark Therapeutics, Inc.

Raw materials for ATMPs can be a unique challenge as some of the materials we work with may be from single sources, may come from non-GMP facilities and process, and may have an evolving supply chain. How do we help our organizations develop a strategy to navigate these challenges from development through commercialization while leveraging our quality systems, material controls, and risk assessments? This session will cover industry examples of approaches to raw materials, from sourcing through testing and release in accelerated environments. 

13:30 – 15:00 | P7: Manufacturing Process Design and Implementation: A Risk-Based and Science-Driven Approach
Moderator: Darius Pillsbury, Senior Consultant, ValSource, Inc.

This session will focus on the design of novel processes for the manufacture of ATMPs and the many challenges encountered in the implementation and monitoring of those processes through the product lifecycle. The employment of science-driven and risk-based approaches to the management of these processes including considerations of the unique quality and technical challenges that must be addressed through clinical and then commercial manufacturing stages will be explored, as well as the many factors that are important to the development of successful control strategies.           

15:45 – 17:00 | P8: Panel of Experts
Moderator: Stephan O. Krause, PhD, Executive Director, AS&T, CT Global Quality, Bristol Myers Squibb

Were there questions you didn’t have a chance to ask during the past two days? Now is your chance to get them answered! Join us for our final panel discussion to hear directly from our industry and regulatory experts.