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Our comprehensive education program offers cutting-edge technical sessions, shedding light on the latest advancements in the pharmaceutical industry.
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Advanced Therapy Medicinal Products
Mon, 14 Oct
Tue, 15 Oct
1000 – 1030
Lifecycle Management Considerations for C&G Therapy
Advanced Therapy Medicinal Products
Regulatory Representative (Invited)
Lifecycle Management Considerations for Cell And Gene Therapy Products to Expand Access And Drive Affordability
Join us as we delve into the realm of lifecycle management considerations for cell and gene therapy products. In this enlightening presentation, the audience will learn more about regulatory strategies aimed at broadening patient access while fostering affordability in the realm of transformative medical innovations. The presentation will illuminate the challenges and opportunities inherent in the lifecycle management of cell and gene therapy products. From initial development stages to post-market considerations, attendees will gain invaluable insights into the regulatory pathways crucial for navigating the dynamic healthcare ecosystem.
Join us as we delve into the realm of lifecycle management considerations for cell and gene therapy products. In this enlightening presentation, the audience will learn more about regulatory strategies aimed at broadening patient access while fostering affordability in the realm of transformative medical innovations. The presentation will illuminate the challenges and opportunities inherent in the lifecycle management of cell and gene therapy products. From initial development stages to post-market considerations, attendees will gain invaluable insights into the regulatory pathways crucial for navigating the dynamic healthcare ecosystem.
1030 – 1100
Navigating Regulatory and CMC Challenges in the Transition
Advanced Therapy Medicinal Products
The development and commercialization of ATMPs present unique challenges at the intersection of regulatory compliance and CMC processes. As companies progress from clinical trials to commercial production, they encounter many regulatory hurdles and CMC complexities that demand strategic navigation for successful market entry. This presentation delves into the critical regulatory and CMC considerations for ATMPs throughout clinical development. We explore key regulatory frameworks, such as FDA and EMA guidelines, and highlight pivotal CMC factors including scalability, process validation, and supply chain management. By addressing these challenges proactively, companies can optimize their development pathways, mitigate risks, and ultimately deliver innovative ATMPs to patients in need.
1100 – 1130
ATMP Regulatory Collaboration & Organizational Readiness
Advanced Therapy Medicinal Products
Jon Halling, Catapult Cell and Gene Therapy
Alfred Penfold, PM Group
Over the last decade, Cell & Gene Therapy Catapult in the UK has worked with industry in the development, production and adoption of ATMPs. CGT Catapult is an independent innovation and technology organization committed to the advancement of cell and gene therapies, with a vision for a thriving industry delivering life-changing advanced therapies to the world. The collaboration with regulators, a total of 15 agency visits / inspections to date, has been a key contributing factor to the success of the Cell & Gene Therapy Facility. Learn how multiple ATMP modalities have been successfully accommodated in the same facility and the steps taken to remain fully compliant.
1300 – 1330
ISPE Guide: ATMPs – Allogeneic Cell Therapy
Advanced Therapy Medicinal Products
Ian Moy, Catalent Pharma Solutions
Komal Hatti, IPS
This discussion is adapted from the 2024 ISPE Allogeneic Cell Therapy Guide, which focuses on manufacturing facility development and design for allogeneic cell therapies. The development, regulatory path, facility design, qualification, and manufacture of ATMPs present significant challenges to manufacturers, engineers, and suppliers. The GMP regulations are evolving as novel processes are presented and manufacturing paradigms are being tested. This discussion addresses facility engineering issues that are most applicable in facility design today, as allogeneic cell therapy manufacturing processes – and ATMPs in general – continue to rapidly evolve.The first section will discuss contamination control strategies and multi-product processing.The second section will discuss the allogeneic cell therapy manufacturing process.The third section will discuss allogeneic cell therapy facilities, key design elements, and common challenges.
1330 – 1400
Lessons Learned - Flexible Cell Therapy Facility Designs
Advanced Therapy Medicinal Products
Andrew Applebury, Fujifilm Diosynth Biotechnologies
George Todorov, IPS
With the number of cell and ex-vivo gene therapy products entering the clinic growing exponentially, we must, as an industry, look ahead and plan accordingly so that we can deliver these revolutionary medicines to large patient populations safely and efficiently. Regardless of the specific modality – and the autologous or allogeneic format – cell therapy processes are not standardized and present significant challenges to facility designs intended to support transitions towards process closure and scale-up, multiple products and/or multiple clients. This presentation will share lessons learned from recent flexible cell therapy facility design projects that enable organizations to meet the needs of evolving cell and ex-vivo gene therapy manufacturing processes.
1400 – 1430
Protecting our Patients, Protecting our Planet
Advanced Therapy Medicinal Products
Maya DeHart, Clark, Richardson, Biskup
AstraZeneca is redefining industry norms and setting a benchmark for sustainable practices on a global scale. A holistic look at AstraZeneca’s global sustainability policy and approach that integrates environmental responsibility, patient-centricity, and operational excellence in cell therapy manufacturing. In this presentation, we will highlight a case study – AstraZeneca’s new cell therapy manufacturing facility in Maryland, which serves as a prime example of the company's sustainability efforts in action. The facility design incorporates an array of features into the design, including 100% electrification, designed for LEED Platinum certification, water reclaim, waste handling, and innovative energy-efficient technologies. The presenters will demonstrate site-specific examples on how to minimize environmental impact while maximizing operational efficiency in cell therapy manufacturing. Lastly, the presentation will explore how sustainability initiatives connect to the resiliency and continuity of supply chain. The discussion will encompass various aspects, including facility design choices, operational readiness, and the company's overarching commitment to patient care. The presenters will elaborate on the strategies implemented for this project, emphasizing this facility’s role in maintaining cell therapy supply chain integrity.
1530 – 1600
Phase & Risk Based Approaches to Enhance ATMP Validation
Advanced Therapy Medicinal Products
Biana Torres, Encoded Therapeutics
Ryan Murray, Valsource Inc
Per Alliance for Regenerative Medicine, there are almost 2000 ongoing clinical trials with an ATMP therapeutic focus as of Q4 2023. The industry often speaks about using risk based approaches to ensure that ATMP products are produced ensuring patient safety, product quality while enabling speed to market. There is a lack of guidance for the framework of risk-based validation in autologous and allogeneic ATMPs. This lack of guidance is leading to a slowdown in clinical trial approaches and commercialization of these novel therapies. This presentation will cover the phase appropriate and risk based approaches specific to process, analytical, cleaning, and supply chain validation strategies that are unique to ATMPs. This presentation will give a preview and provide some examples from an ISPE ATMP Guide covering this topic that is expected to be issued either before or shortly after Oct 2024.
1600 – 1630
CDMOs—Swift Patient Access with CQV & Project Management
Advanced Therapy Medicinal Products
Robin Newman, Core Services Group
Gretchen Smith, Advanced Medicine Partners
CDMOs have taken over the pharmaceutical field especially when it comes to development of novel therapies such as cell therapies, gene therapies, and RNA products which are critical to the patient population facing rare diseases. Advanced Medicine Partners has shifted the paradigm that CDMOs should work independently from clients and has taken the approach to partner with clients in various phases of development, including providing an extended workbench as a novel offering. ATMPs can leverage AMP’s wide range of analytical, process optimization and development, and GMP manufacturing capabilities to accelerate successful drug candidates getting to patients.This presentation proposes to delve into the relationship of client-partnerships, CQV deliverables, and project management tools that impact the flexibility and speed of a CDMO. Implementation of efficient site procedures with project controls over CQV deliverables can facilitate seamless tech transfers. The presentation will go over how to build a robust CQV plan, which plays an important role in the life cycle as it demonstrates product integrity and regulatory compliance. The presentation will demonstrate how using proper CQV & project management tools to manage resources, timelines, and deliverables helps not only to deliver quality products faster-to-market but can help control costs and optimize spending.
Gretchen Smith is a highly accomplished manufacturing executive with an impressive track record spanning over 25 years in the pharmaceutical industry. Currently serving as the Senior Vice President of Manufacturing and Site Head at Advanced Medicine Partners, she plays a pivotal role in leading the establishment of a cutting-edge Contract Development and Manufacturing Organization (CDMO) facility. This state-of-the-art facility is dedicated to providing unparalleled chemistry, manufacturing, and controls (CMC) capabilities for advanced medicines, including gene and cell therapies. Gretchen's expertise allows her to address the critical challenges of product quality and scalability that are often encountered in the development and production of highly technical medicines.
Gretchen Smith is a highly accomplished manufacturing executive with an impressive track record spanning over 25 years in the pharmaceutical industry. Currently serving as the Senior Vice President of Manufacturing and Site Head at Advanced Medicine Partners, she plays a pivotal role in leading the establishment of a cutting-edge Contract Development and Manufacturing Organization (CDMO) facility. This state-of-the-art facility is dedicated to providing unparalleled chemistry, manufacturing, and controls (CMC) capabilities for advanced medicines, including gene and cell therapies. Gretchen's expertise allows her to address the critical challenges of product quality and scalability that are often encountered in the development and production of highly technical medicines.
1630 – 1700
Case Study - rAAV - Changing Bioreactor Models / ScaleUp
Advanced Therapy Medicinal Products
Apurv Puri, Pharmatech Associates
The gene therapy industry has witnessed comparability issues manifest throughout the clinical trial phases. While these issues were not attributed to process changes per se, they highlight the lack of thorough process understanding and process robustness when manufacturing novel products. AAV vectors are not considered “well characterized” products. This creates a challenging landscape for demonstrating comparability - and with rapidly evolving technology and manufacturing platforms, there is a need to drive systematic approaches to equipment selection and evaluation. The ISPE ATMP CoP has recently published a guide on rAAV Comparability to bridge this gap. This session will present a case study on demonstrating rAAV product comparability when changing the production bioreactor configuration and scaling up.
1015 – 1045
Advances Needed to Expand Cell & Gene Therapies Adoption
Advanced Therapy Medicinal Products
The breadth of Daniel's perspective in the Cell and Gene Therapy space encompasses early research laboratories purchasing Lonza's media, tool & technologies as well as large biotechs and big pharma, outsourcing commercial manufacturing to Lonza. During this address, Daniel will reflect on where the field stands and on the sequence of development and CMC innovations necessary for cell and gene therapies to become truly mainstream and adopted as the standard of care for a wide range of indications and large patient populations. Further to this, Daniel will reflect on the potential futures of cell and gene therapies, depending on a range of potential scenarios.
1045 – 1115
Decentralized Pharma
Advanced Therapy Medicinal Products
Decentralized Pharma: Pioneering Patient-Centric Manufacturing
Background: Germfree, an innovator in biosafety and containment technologies, has been at the forefront of deploying more than 100 mobile cleanrooms in seven years. Challenge: Healthcare faces ongoing challenges in patient access, drug shortages, manufacturing inefficiencies, and the need for rapid response to patient needs. Traditional centralized manufacturing models often struggle to adapt quickly to these demands, creating a pressing need for innovative solutions.Solution: Germfree responded to this challenge by developing and deploying advanced mobile cleanroom technologies. These units are designed to be flexible, scalable, and capable of meeting the stringent requirements for aseptic processing and manufacturing of cell and gene therapies, as well as other pharmaceutical products.Implementation: The case study will detail the deployment process of these mobile cleanrooms, including site selection, logistics, setup, and integration into existing healthcare and pharmaceutical frameworks. Lessons Learned:Future Outlook: Looking ahead, Germfree's work signals a shift in pharmaceutical manufacturing, highlighting the potential for decentralized models to make healthcare more accessible, efficient, and patient-centered. The case study will conclude with predictions for the industry and the role of mobile cleanroom technology in shaping the future of pharmaceuticals.
Background: Germfree, an innovator in biosafety and containment technologies, has been at the forefront of deploying more than 100 mobile cleanrooms in seven years. Challenge: Healthcare faces ongoing challenges in patient access, drug shortages, manufacturing inefficiencies, and the need for rapid response to patient needs. Traditional centralized manufacturing models often struggle to adapt quickly to these demands, creating a pressing need for innovative solutions.Solution: Germfree responded to this challenge by developing and deploying advanced mobile cleanroom technologies. These units are designed to be flexible, scalable, and capable of meeting the stringent requirements for aseptic processing and manufacturing of cell and gene therapies, as well as other pharmaceutical products.Implementation: The case study will detail the deployment process of these mobile cleanrooms, including site selection, logistics, setup, and integration into existing healthcare and pharmaceutical frameworks. Lessons Learned:Future Outlook: Looking ahead, Germfree's work signals a shift in pharmaceutical manufacturing, highlighting the potential for decentralized models to make healthcare more accessible, efficient, and patient-centered. The case study will conclude with predictions for the industry and the role of mobile cleanroom technology in shaping the future of pharmaceuticals.
1115 – 1145
The Matrix Approach: Revolutionizing Aseptic Filling?
Advanced Therapy Medicinal Products
Holger Kranenburg, KyooBe Tech GmbH
Patrick Wieland, Bausch+Ströbel SE + Co. KG
In this presentation we explore a visionary approach to aseptic fill and finish processes. With operational flexibility based on technical solutions, we provide an answer to challenges deriving from diversified product portfolios and changing pipelines. The approach is applying highly standardized robotic isolator cells addressing the growing demands for highest quality as well as contamination and cross contamination control. In the matrix framework all modules are combined into a versatile functional unit to deliver efficiency, flexibility and reliability at a new level.The concept is applied as a suggested production layout that will highlight the matrix approach benefits in comparison to a conventional layout. This layout is designed to efficiently handle diverse requirements, from fill and finish of very small batch applications like personalized ATMPs to mid-scale production of conventional biologics (e.g. 25.000 units/batch).
1530 – 1700
Expediting Affordable & Available Therapies for Patients
Advanced Therapy Medicinal Products
James Wabby, AbbVie, Inc.
Erich Bozenhardt, United Therapeutics
Peter Millili, Johnson & Johnson
In the rapidly evolving field of cell and gene therapy manufacturing, companies are grappling with significant cost pressures as they strive to bring groundbreaking therapies to market. A critical factor influencing these costs is the selection of manufacturing technologies, which directly impacts facility design requirements, including the size and specification of cleanroom environments. Automation is recognized as a tool to enable a combination of scalability, reduced manufacturing costs and increased quality. This panel aims to discuss the technical, quality, and regulatory considerations associated with expanding access of these therapies.
Speaker Qualifications
Speakers selected to present at ISPE events are leading professionals in their fields. However, it may be necessary to make substitutions. Every possible effort will be made to substitute a speaker with comparable qualifications. Every precaution is taken to ensure accuracy. ISPE does not assume responsibility for information distributed or contained in these events, or for any opinion expressed.
Programme Changes
Programme is subject to change. Last minute changes due to functional, private, or organisational needs may be necessary. The event organiser accepts no liability for any additional costs caused by a change of programme.