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Advancements in Cell and Gene Therapy Manufacturing

Francesco Cicirello
Ryan Paul Mazur
Advancements in Cell and Gene Therapy Manufacturing

In the wake of the COVID-19 pandemic, the landscape of cell and gene therapy (CGT) manufacturing has witnessed significant transformation. The industry has seen an increased emphasis on the advantages of advanced therapy medicinal products (ATMPs) and the push for increases in manufacturing scale and overall availability to larger populations. ATMPs find themselves at a critical juncture where the intricate nature of personalized medicine necessitates advancement in manufacturing capabilities to meet the rising demand and ensure patient access to these cutting-edge therapies.

The 2024 ISPE Aseptic Conference Planning Committee is proud to present the track Production Case Studies / ATMPs. This track will highlight a combination of production case studies and specific technical presentations centered around innovative technologies and manufacturing strategies to achieve increased output of ATMPs. Discover the captivating themes below that have been carefully curated for the 2024 ISPE Aseptic Conference being held in Vienna, Austria from 12-13 March 2024.

Flexibility in GT Manufacturing:

In order to reveal the full potential of future therapies, manufacturing processes must be designed for future flexibility. Flexibility is best defined as allowing scalability and adaptability to reach different patient populations while employing the highest of technological advancements. The session How to Approach the Deliverable of Flexibility Demanded by Lizette Janse Van Rensburg, Head of Production, PCI Pharma Services, will highlight how flexibility is needed in the manufacturing process of autologous and allogeneic therapies to meet the growing demand.

Aseptic Fill/Finish Innovations and Considerations:

The subsequent increased demand for adaptable and versatile CGT manufacturing processes has shown a pivot shift in aseptic filling technology to accommodate diverse production requirements for various CGTs and ATMPs. Advanced technologies have emerged for cleaning and sterilization processes to mitigate the risk of cross-contamination between different pharmaceutical products. The presentation Next Generation of Aseptic Filling According to Annex 1 by Patrick Wieland, Global Lead Business Development, Bausch and Ströbel, will highlight advancements in the hygienic design standards of aseptic filling lines ensuring product integrity and patient safety. The presentation will be supported by a case study of Sanofi’s new aseptic filling line and how the design of the line prioritized flexibility and adherence to the highest quality standards for cleanability and sterilization according to updated Annex 1 regulations.

Aseptic Filling has also seen an increase in technological innovations and options available due to the updated regulations of Annex 1. Operating companies have witnessed an increase in the number of different containers that therapy products and ATMPs can be filled in prompting business decisions on whether to use multi-use multi-format machines or traditional machines with various fill volumes for the same container. Other important considerations include environmental monitoring, deciding between traditional agar settling plates with higher aseptic interventions or continuous viable particle monitoring with lower aseptic interventions. In their presentation Innovations on New CMO Aseptic Filling Line, Marc Machauer, Sales Manager, Particle Measuring Systems GmbH, and Rainer Gloeckler, Swissfillon AG, will highlight sought-after technological innovations driven by Annex 1 regulatory requirements and business drivers for Ten23 Health.

Transformative Organizational Change for Cell Therapy Delivery:

The difficulty of delivering cell therapies to a large number of patients has revealed how traditional functional groups for operating companies impede manufacturing and quality operations. The complex manufacturing process of cell therapies has inherently created bottlenecks, highlighting the need for organizational shifts and changes to prioritize collaboration and integration among functional groups within organizations. Mike Kelliher, Executive Director, Cell Therapy Operations, at Bristol Myers Squibb (BMS), will highlight the paradigm shift that BMS embraced at its site in Summit, New Jersey, USA to seamlessly manufacture two commercially approved cell therapy products. His presentation Cell Therapy: BMS’ Value Stream Transformation will delve into the innovative value stream strategies BMS implemented to break down traditional barriers in the cell therapy manufacturing process, fostering collaborative integration of organizational functional groups to facilitate expedited product turnaround times and sustained improved delivery times.

Maximizing Efficiency and Yield for Gene Therapies:

The complex nature of gene therapy manufacturing typically involves intensive manual manufacturing operations that can lead to product loss and increased costs of goods sold (COGS). In the manufacturing process, the fill/finish step is critical to ensure optimal drug product availability. Operating companies are realizing the heightened importance of aseptic filling systems that reduce manual labor, mitigate contamination, employ precision with pump technologies, and implement real-time data monitoring for production parameters. In their presentation Maximizing Gene Therapy Doses for Patients, Scott Sznyter, Associate Director - Drug Product Manufacturing and Cell Banking, Center for Breakthrough Medicines, and Dena Flamm, Business Development Manager, OPTIMA Pharma, will highlight how the Center for Breakthrough Medicines addressed challenges with gene therapies to preserve their efficacy and integrity by leveraging Optima’s FillCell technology.

Point-of-Care ATMP Manufacturing:

Aseptic processes for ATMPs have traditionally been centralized and tailored to specialty product formulations for individual patients. Such formulations can require final aseptic manipulations such as cell washing or reconstitution before administration to patients. Due to the aseptic processing and final transport logistics, there is a heightened interest in point of care manufacturing for ATMPs. Having the capability of performing aseptic techniques and processes while eliminating distance to the patient and time to treat the patient could greatly reduce therapy costs. The presentation Maintaining Control in Point of Care Aseptic Processing for ATMPs by Krisha Patel, Partner, Assurea LLC, Carol Houts, Vice President of Quality and Regulatory, Germfree Laboratories, Inc., and Ohad Karnieli, Pluristem Therapeutics, will highlight the drivers for ATMP manufacturing at the point of care and explore the regulatory framework to implement such manufacturing at the point of care for critically ill patients.

As the presentations and descriptions above highlight, there has been a significant change and development in the field. The rise of prominence for ATMPs and novel therapies speaks of their evolution beyond mere CAR-T products. Recent regulatory authority approvals for treating serious diseases such as hemophilia or sickle cell sickness further corroborate this trend. Yet, despite the demonstrated potential to cure the incurable, the profitability of these novel therapies presents somewhat of a conundrum. While production cost is not the sole determinant of price and profitability, it undeniably plays a crucial role.

Addressing these profitability challenges is precisely what the presentations above aim to achieve: An overarching view of various challenges around ATMP production, including manufacturing platform, manufacturing scale, aseptic fill/finish innovations and advancements, supply chain resiliency, and the latest impacts of updated Annex 1 regulations related to process design and aseptic filling.

If you are looking to explore recent innovations, address ongoing challenges, and collectively advance the field of ATMPs, this conference is for you. We invite you to join us in Vienna, Austria for the 2024 ISPE Aseptic Conference, where these crucial discussions will unfold. Your participation is vital as we collectively advance the field of ATMPs. Come and contribute to the discourse!

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