Agenda

Learning Objectives

  • Explore case studies in process development and equipment design for profitable and sustainable manufacturing. 
  • Gain awareness of the changing landscape of cell and gene therapy development, testing, and commercialization. 
  • Learn about innovations in facility design and operations, and strategies for collaboration between manufacturers and regulators. 
  • Implement best practices used by CDMOs in non-traditional biopharma processes. 
  • Understand the supply chain risks and challenges associated with the production of personalized medicines.

Topic Areas Include

  • Cell and Gene Therapies from Research to Clinical to Production
  • Emerging Technologies to Deliver Cell and Gene Therapies
  • Supply Chain Challenges and How the Industry is Meeting Them (including facility design & operations)
  • Aspects of Automation and Digitalization in BioManufacturing
  • Challenges facing vector-based gene therapies
  • Harmonization: Regulatory, CMC, GMP

All of the agenda times below are displayed in EDT.

Day 1
Monday, 1 June 2020
  • 0900 - 1030
    Opening Plenary - Challenges and Opportunities in the Manufacturing of New Treatment Modalities
    This year, the 2020 ISPE Biopharmaceutical Manufacturing Conference will focus on advances in manufacturing, facilities, and analytics important to bring new therapies to patients. Now more than ever, manufacturing is on the critical path as many new treatments in the area of personalized medicines and individualized batch production present the challenge to bring these medicines through rapid development, reliable manufacturing, and availability to patients a pace with the advances clinical research is bringing to address unmet needs. At the same time, biosimilars are advancing with the opportunity to provide more access to medicines.  In this opening session, we will hear an important regulatory perspective from FDA on gene therapy drug development and a reflection from industry on 10 years of Biosimilars.
    Session Leaders

    Joseph C. Famulare

    Vice President, Global Quality Compliance and External Relations
    Genentech, A Member of the Roche Group
    Speakers

    Carol Lynch

    President, Sandoz US, Head, North America
    Head, North America
    Sandoz US Inc.

    Eric D. Felz

    Associate Director Validation
    Takeda Pharmaceutical Company Limited
    Presentation Information
    [Gene Therapy: Efficient Drug Development]
    Speaker: Wilson Bryan
    || [Reflecting on a Decade with the Biologics Price Competition and Innovation Act]
    Speaker: Carol Lynch
    This year marked the ten-year anniversary of the BPCIA. As the industry reflects on a decade of experience with the US biosimilar pathway, this session offers an opportunity to reflect on the growing momentum for biosimilars in the US, and discuss issues central to the BPCIA that remain to be resolved.
  • 1030 - 1045
    Break
  • 1030 - 1830
    Virtual Exhibit Hall Open
  • 1045 - 1215
    Concurrent Session - Planning, Building and Operating Cell and Gene Therapy Facilities (Part I)
    Many cell and gene therapy facilities are in different phases of planning, construction, and expansion.  Learn from experts in engineering and manufacturing about how these facilities are progressing.   This two-session track will explore various designs as we move from lab scale to higher volume production; control for single use to allow for multiple manufacturing trains. These sessions will examine going from manual to automated processing to meet the demand and the various technologies and layouts being utilized to supply patient needs and meet regulatory GMP requirements.
    Session Leaders
    Speakers
    Presentation Information
    [Building Flexibility for Multiple Cell Therapy Processes]
    Speaker: Daniel Swanson
    Many cell therapy firms have a diverse pipeline of promising drug candidates and are faced with the growing challenge of how to manufacture these drugs in a safe, timely and cost-effective manner. Failed clinical trials leave abandoned assets behind, but manufacturing multiple products in the same facility can mitigate much of the associated risk costs. To manufacture multiple cell therapy products safely, deliberate strategy needs to be implemented from the inception of facility design. Product segregation, biological safety level boundaries, small (many times one patient) batch sizes, operator safety, and flexibility for manual manipulations. This presentation will assess the best practices and case studies of building flexibility of multiple products in the same facility and weigh the benefits and costs of the flexibility.
    || [Leveraging Operational Simulations for Gene Therapy Facilities]
    Speaker: Niranjan Kulkarni
    Cell therapies are emerging as an important class of next-generation medicines. As many novel treatments move through development to commercialization, manufacturers are challenged to design and construct optimal facilities for the production of cell therapies. Designing optimal facilities and operations also has a significant impact on the Cost of Goods. Computer modeling and simulations from an operational perspective can support the development of an optimal facility design along with developing operating strategies that will enable cell therapy manufacturers to meet the needs of patient populations, while better managing the Cost of Goods. This presentation will focus on innovation in technologies that enable a competitive and sustainable biopharmaceutical product supply for the future.
    || [Viral Vector Manufacturing in a Traditional Biopharm Plant ]
    Speaker: Edward Stevens
  • 1045 - 1215
    Concurrent Session - Pharma 4.0: What it is, What it isn’t and How to Implement it
    Trying to figure out what Pharma 4.0 is and what it means to your business? This session will clarify what Pharma 4.0 is and offer practical aspects of how it has been implemented in the plant and what it means to your workforce.
    Session Leaders

    Eric D. Felz

    Associate Director Validation
    Takeda Pharmaceutical Company Limited
    Speakers

    Patrick Boyle

    President and CEO, Skillpad Canada Inc.
    SKILLPAD Canada Inc.

    Shin Kawamata, MD, PhD

    Director of R&D Center for Cell Therapy
    Foundation for Biomedical Research and Innovation At Kobe
    Presentation Information
    [Pharma 4.0 Meets Operator 4.0: Facing Knowledge Challenges of Manufacturing in the 4.0 Era] Speaker: Patrick Boyle
    || [Enabling Automation and Pharma 4.0 in Cell Therapy]
    Speaker: Shin Kawamata
    || [Why Pharma 4.0? Did We Advance?]
    Speaker: Christian Woelbeling
    Pharma 4.0 is the holistic approach in the supply chain to make the ICH Control Strategy happen in manufacturing environments. We need the interdisciplinary collaboration of different departments to design a robust and flexible production execution environment. This presentation will discuss how to approach a Holistic Control Strategy implemetnation, its benefits when it is designed with a cross-divisional approach, and what we achieved in the ISPE industry initiative Pharma 4.0 so far. Based on best practice case studies, the enablers, elements, and challenges of the Holistic Control Strategy implementation will also be discussed.
  • 1215 - 1245
    Lunch Break
  • 1245 - 1415
    Concurrent Session - Planning, Building and Operating Cell and Gene Therapy Facilities (Part II)
    Many cell and gene therapy facilities are in different phases of planning, construction, and expansion.  Learn from experts in engineering and manufacturing about how these facilities are progressing.   This two-session track will explore various designs as we move from lab scale to higher volume production; control for single use to allow for multiple manufacturing trains. These sessions will examine going from manual to automated processing to meet the demand and the various technologies and layouts being utilized to supply patient needs and meet regulatory GMP requirements.
    Session Leaders
    Speakers
    Presentation Information
    [The Impact of Innovation on Building the Facilities of the Future]
    Speakers: John Khoury & Alexis Melendez
    This presentation will address the challenges of creating a modern cell therapy manufacturing facility in the midst of constant change. Presenters will discuss utilization of new technologies and techniques in the cell therapy field to forge the path for the next generation of biopharmaceutical manufacturing. Adaptability, flexibility, and ingenuity are the cornerstones necessary to cross over the challenges that an innovative facility will inevitably face. As a result of this presentation, participants will gain an understanding of lessons learned in the following areas: maintaining adaptability to overcome challenges while not losing sight of the envisioned goal, prioritizing flexibility to adjust approaches in adversity, and encouraging ingenuity to utilize cutting-edge equipment and techniques to create a modern facility.
    || [ATMP Facility Scale-up to Production using Isolator Technology]
    Speaker: Michael Hennessy
    As the ATMP product portfolio transfers through the research pipeline to latter stage clinical and commercialisation, the emphasis turns to the design of FDA/EMA compliant, BSL-2 enabled multi-product processing within cGMP environments with scalable equipment such as isolator technology. The risk of product contamination by operators, and cross contamination across different product streams, further reinforces the necessity to transition early to ‘closed’ processing systems. This presentation will outline how isolators can facilitate operational ‘scalability’; reduce overall building footprint and mitigate against potential contamination concerns. We will review how process equipment, often designed for open laboratory processing, can be successfully integrated into closed isolator systems and consider future developments with respect to isolator design and robotics.
  • 1245 - 1415
    Concurrent Session - Application of ICH Q12 Principles in Modern CMC Submissions
    This session brings together industry and regulatory agency representatives to discuss case studies involving the implementation of ICH Q12 principles in regulatory submissions to facilitate post-approval CMC changes in an efficient and predictable manner.
    Session Leaders
    Speakers

    Andrew Chang, PhD

    Vice President, Quality and Regulatory Compliance, Product Supply Quality
    Novo Nordisk
    Presentation Information
    [Application of ICH Q12, Established Conditions, and a Product Life Cycle Management Document to the Analytical Aspects of the 2019 FDA Q12 Pilot Program]
    Speaker: Michael Cohen
    || [FDA Perspective on the Implementation of ICH Q12]
    Speaker: Terrence Ocheltree
    || [ICH Q12 and Beyond: The Journey Towards Regulatory Convergence in Product Life Cycle Management]
    Speaker: Nina Cauchon
    || []
    Panel Discussion:
    Andrew Chang
    Michael Cohen
    Nina Cauchon
  • 1415 - 1430
    Break
  • 1430 - 1600
    Concurrent Session - Cell and Gene Therapy CMC and Manufacturing: Ensuring the Analysis, Production and Quality
    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.
    Session Leaders
    Speakers

    James Warren

    VP, Pharmaceutical Development
    Ultragenyx Pharmaceutical Inc.
    Presentation Information
    [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.
  • 1430 - 1600
    Concurrent Session - Technology Advancements for Drug Product Manufacturing
    This session will discuss technology advances related to the manufacturing of biologic drug products of different dosage forms.  Technologies and case studies to be discussed include low temperature sealing of container closures and continuous aseptic spray freeze-drying.
    Session Leaders
    Speakers

    Laura Moody, PhD

    Product Manager – Primary Packaging, North America
    Syntegon Pharma Technology

    Joshua Stauffer

    Director, Drug Product Manufacturing System Design & Commercialization
    Merck & Co., Inc.
    Presentation Information
    [Dynamic Challenge of Sealing Performance for Biologic Container Systems at Cold Temperature ]
    Speaker: Qingyu Zeng
    It has been well known to biopharmaceutical manufacturing that it is very challenging to obtain and maintain sealing performance of container system at low temperature. The significant loss of sealing performance at lower temperature transitions could potentially contribute significant risk to drug product integrity at low storage and transport temperatures. This presentation demonstrates that container sealing performance will change inherently by itself during temperature transition in real time and over time, and will address: (1) what negative impact happens to sealing performance of biopharmaceutical container system at low temperature; (2) why this negative impact happens; and (3) how to mitigate the loss of sealing performance at lower temperature through biopharmaceutical manufacturing. The presentation provides a new, integrated methodology framework and fresh acumen to the biopharmaceutical manufacturing for practically and proactively considering, designing, setting up, controlling, and managing container stopper sealing performance risk.
    || [Single-Use Technology for Final Fill: An Overview]
    Speaker: Laura Moody
    || [Vial Visualization Using AI]
    Speaker: Joshua Stauffer
  • 1600 - 1615
    Break
  • 1615 - 1745
    Concurrent Session - Increased Automation and Digitalization in Manufacturing
    Computer systems have continued to help manage complexity involved in biopharmaceutical manufacturing.  Such systems have evolved in ways previously not anticipated.  This session will explore some of the evolving uses of computer systems to allow a greater focus on product quality.
    Session Leaders

    David Doleski

    Compliance Head for Biologics Quality Operations
    Sanofi
    Speakers

    Martin Mayer

    Business Development Smart Engineering Services & Digitalization
    ZETA GmbH
    Presentation Information
    [Integrated Engineering the Key to Cost Effective Digitalization]
    Speaker: Martin Mayer
    Recently, in the pharmaceutical industry, a number of key business drivers have been identified – agility, flexibility and adaptability – that are required to meet the ever-increasing demands of reduced time to market, personalized medicine, improved cost effectiveness and best-in-class competitiveness. Digitalization has introduced new trends and technologies. Better trained employees, flexible production, and smart maintenance strategies are seen as cornerstones to success - and the Digital Twin is often seen as the tool for realization. Retrieving information about the “as built” status of a plant and re-engineering the Digital Twin is costly and time consuming. Creating the Digital Twin in engineering phase by integrating all disciplines within ONE toolchain delivers in best in class results. The presentation will introduce a categorization of DigitalTwin with respect to pharma 4.0 applications, including virtual commissioning, predictive maintenance, operator training, startup trainings, schedule/batch optimization, examples of Digital Twin applications, and an outlook of what to expect within the next years.
    || [How Digital is Transforming Quality Assurance Operations: A Focus on Real and Right Time Release]
    Speaker: Michael Shanno
    || [BioPhorum Plug and Play: Developing and Utilizing Standards to Enable Reduced Project Delivery Time]
    Speaker: Bruce Kane
    The ability to quickly deploy and replicate standard production systems worldwide will be critical in the deployment of a worldwide vaccine for the severe acute respiratory syndrome coronavirus 2(SARS-CoV-2. The presentation will also discuss the dependencies to the Namur MTP 2568 standard as well as to OPC/UA. The presentation will also describe two completed proof of concepts to test out the concepts and principles developed. A third POC is being planned.
  • 1615 - 1745
    Concurrent Session - Practical Challenges with Quality System Management in Cell and Gene Therapies
    This session will focus on the challenges with the establishment of quality oversight and systems from the initial patient interface through manufacturing and supply for cell and gene therapies.  Experience with the application of cGMPs in the hospital setting will be discussed as well as the conflicting priorities when multiple companies are imposing different demands on the same institutions, notably as these controls are part of the approved applications. The efforts to harmonize standards globally, as well as the regulator experience in determining true compliance risks will be addressed.
    Session Leaders
    Speakers

    Lina Ertle

    Head of External Relations EMEA and Japan
    F. Hoffmann-La Roche Ltd.
    Presentation Information
    [Quality Considerations for Cell & Gene Therapy]
    Speaker: Luciana Mansolelli
    || [FDA Perspective on GMPs for Cell & Gene Therapies]
    Speaker: Ekaterina Allen
    || [Challenges for GMPs for ATMPs: European Perspective]
    Speaker: Lina Ertle
  • 1745 - 1830
    Virtual Networking Session for Attendees, Speakers, and Exhibitors
Day 2
Tuesday, 2 June 2020
  • 0900 - 1030
    Regulatory Updates
    Plenary Session - Regulatory expectations related to biotechnology and biological products are continually evolving both within mature and emerging regulatory agencies. This session will provide a snapshot of recent regulatory changes across the global landscape. Invited speakers will provide a sampling of some of the recent regulatory responses related to the ongoing COVID 19 pandemic, discuss the rapidly evolving regulatory landscape in China, and highlight some of the recent trends related to sterile products in the United States.
    Session Leaders
    Speakers

    Joseph C. Famulare

    Vice President, Global Quality Compliance and External Relations
    Genentech, A Member of the Roche Group
    Presentation Information
    [Inspectional Trends for Biotech Products and Drug Substance]
    Speaker: FDA Representative Invited
    || [Update on Sterile Products and Change Management]
    Speaker: FDA Representative Invited
    || [Regulatory Requirement Differences Between China and US for Biologics & Biosimilars]
    Speaker: Lawrence Hill
    || [Update on Sterile Products and Change Management]
    Speaker: Richard Friedman
    || [COVID 19 Regulatory Update]
    Speaker: Joseph Famulare
  • 1030 - 1045
    Break
  • 1030 - 1730
    Virtual Exhibit Hall Open
  • 1045 - 1215
    Concurrent Session - Automation, Robotics and Digitalization in Biopharmaceutical QC Labs
    The goal of this session is to engage laboratory leaders, automation and informatics experts, regulators, and instrument/equipment/software vendors in rich discussions regarding the use of automation, robotics, and digital solutions to advance multiple dimensions of operational excellence in the pharma quality control laboratories.
    Session Leaders
    Speakers

    Vinny Browning

    QC Global Network Leader and Global Process Owner
    Amgen Inc

    Sven M. Deutschmann, PhD

    Head of Global ASAT Adventitious Agents Testing & Alternative Microbiological Me
    Roche Diagnostics GmbH
    Presentation Information
    [QC Today and QC in the Future]
    Speaker: Vinny Browning
    || [Case Study: Automated Technology Platform for Real-Time PCR-based Adventitious Agent Detection]
    Speaker: Sven Deutschmann
    || [Case Study: Automation of Stability Data Reporting and Trend Analysis]
    Speaker: Janine Kuratli
    || [Technology Solutions & Business Drivers for Moving from Manual to Automated Solutions in the Pharmaceutical QC Lab]
    Speakers: Jacqueline Larew and Mark Schweitzer
  • 1045 - 1215
    Concurrent Session - Single Use Technology Strategies
    The approach to manufacture is never simple and now more and more hybridized. In this session experts share their latest findings and recommendations in SUT.  Given the movement toward personalized medicine even if you are not a single use proponent, getting ready for the future is important to your business
    Session Leaders
    Speakers

    Lindsay Smart

    Business Development Manager UK, Ireland, North America
    ZETA GmbH
    Presentation Information
    [Single-use Versus Stainless Steel Biomanufacturing Facilities: Are Hybrid Facilities the Best of Both Worlds?]
    Speaker: Lindsay Smart
    The pharmaceutical industry is constantly facing severe time and cost pressures. Currently, in order to be able to react to new developments in the sector, as quickly as possible, the focus is ever increasingly centred on time-to-market as well as modular, highly flexible production concepts for new biopharmaceutical products. This presentation will detail: 1) How current assemblies can be customized and interconnected with highly sophisticated cross-functionality from a mechanical and automation perspective; 2) The advantages of next level single-use systems from recent case studies, which will show how dance floor concepts improve process clarity and ergonomics, operator safety and simplify maintenance and 3) Supplier agnostic concepts showing how a stainless steel backbone can handle generic single use bioreactors and other single-use assemblies to optimize facility design and layout.
    || [Going from N-1 to Purified Product in a Closed and Connected Single-use Solution]
    Speakers: Pietro Perrone & Hande Özgen
    This presentation will focus on innovation in production methods and technologies that enable a competitive and sustainable biopharmaceutical product supply. The data presented is obtained from a PD environment and will demonstrate the power and possibilities of closed and connected single use processes. Furthermore, the results presented will prompt discussions on when it’s appropriate to use connected and closed manufacturing. These are important topics in an industry that aims to maximize productivity, flexibility and quality in the manufacture of biopharmaceutical products.
  • 1215 - 1245
    Lunch Break
  • 1245 - 1415
    Preparing for the Workforce Needs in Biopharma Manufacturing
    The new therapeutic modalities and associated manufacturing processes and technologies require a new workforce skillset. Process automation, process intensification, continuous manufacturing, and scale-out versus scale-up are examples of what are becoming new norms in manufacturing. The demand for a trained workforce is challenging many biopharma companies. This session will discuss approaches for meeting these challenges.
    Session Leaders
    Speakers

    Lindsey Silva, PhD

    Senior Manager, Microbiology Global QC Technology
    Genentech, A Member of the Roche Group
    Presentation Information
    [Biopharmaceutical Manufacturing Workforce Development in the Age of Gene Therapy]
    Speaker: John Balchunas
    The community of education, training, and workforce development professionals are keenly focused on emerging technologies such as gene therapy, cell-based therapies, gene editing, and regenerative medicine. Caught up in the race to commercialization, industry and academia are developing new processes, new job classifications, and disruptive technologies that could change the manufacturing paradigm altogether. This talk will 1) take a broad look at the impact of these emerging technologies on the biopharmaceutical manufacturing workforce, 2) profile the gene therapy vector manufacturing workforce and current challenges faced in clinical and commercial-scale manufacturing operations, 3) introduce some of the workforce development initiatives NIIMBL has catalyzed to help begin mitigating gene therapy workforce challenges, and 4) discuss anticipated impacts of gene therapy industry growth on the overall biopharmaceutical manufacturing workforce ecosystem.
    || [University Engagement Successes]
    Speakers: Antonio Moreira, Lindsey Silva, and Manfred Maeder
  • 1415 - 1430
    Break
  • 1430 - 1530
    Plenary Session - Innovation in Technological Platforms in Biopharmaceutical Manufacturing
    As described in the morning session, regulatory expectations continue to evolve given the complexities associated with new products and innovation. This session will provide an opportunity to hear more about this topic in the context of an innovative vaccine facility and related sterile manufacturing requirements.
    Session Leaders
    Speakers
    Presentation Information
    [Process and Facility Innovation using PODs in Vaccine Manufacturing – Benefits and Challenges]
    Speaker: Maria Hoffman
    This presentation will focus on a project that provides an end-to-end filling and packaging operation for a vaccine in a POD (Portable On Demand) facility. Vaccine supply chains are typically very long and require production prior to precise demand signals. In addition, cold chain requirements further complicate the supply chain when global volumes are made at a single location and benefit from a smaller footprint with the flexibility to relocate as needed. The technology used to shorten lead time and the benefits that the POD offers for this and other use cases will be reviewed. The potential technical and regulatory obstacles to portability of a POD will also be addressed.
    || [The Future of Biomanufacturing: Regulatory Perspectives – Changing Landscape ]
    Speaker: Patricia Hughes
  • 1530 - 1545
    Break
  • 1545 - 1645
    Closing Plenary Session & Fireside Chat - Regulatory and Industry Insights & Lessons Learned
    Wrapping up the conference it is important to hear from regulatory leaders to help frame the strategy for the era ahead.  Lessons learned from organizations around the world will give individuals the most current insights for those difficult discussion back at your home base.
    Session Leaders
    Speakers
    Presentation Information
    [Facilitating Development of Advanced Therapies]
    Speaker: Raj Puri

    Industry and Regulatory Fireside Chat Panel:
    Raj Puri
    Patricia Hughes
    John Balchunas
    Lawrence Hill
    Maria Hoffman
    || []
    Panel Discussion: Lawrence Hill, John Balchunas, Maria Hoffman, and Patricia Hughes
    || [Innovations in Sterile Manufacturing – Regulatory Considerations]
    Speaker: Patricia Hughes
  • 1645 - 1700
    Closing Remarks
    Speakers

    Joseph C. Famulare

    Vice President, Global Quality Compliance and External Relations
    Genentech, A Member of the Roche Group