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Cell & Gene Therapy 2018: Scientific Program
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To view the final program, click here.  

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Keynote Presentation - Tuesday, July 10, 2018   

Advancing Manufacturing for Advanced Therapies 
Peter Marks, MD, PhD, Director, CBER, FDA, USA 

Session Abstracts

First-in-Human and Early CMC Development

Session Chair: Ilona Reischl, AGES-Austrian Agency for Health and Food Safety

The progression from preclinical studies to first-in-human clinical trials is a milestone in the development of medicinal products. The goal of this session is to elaborate on specific aspects to consider and challenges to address in enabling for cell- and gene- therapy products and tissue engineering products (Advanced Therapy Medicinal Products, ATMPs) to reach and pass this step.

The definition of active substance and final product is not always as obvious for ATMPs as it is for other products, yet this is the basis for all steps from characterization of starting materials to product requirements and proof-of-concept. The planned mode of administration, including the potential need for tailored administration devices and transport logistics, additionally impact on quality documentation needs. We will discuss how the nature of the product informs the risk-based approach to determine data requirements.

Regulatory requirements between the different regions vary, particularly for first-in-human studies. What is required for phase I and what should be done to facilitate later development? The topics will be elaborated with overview presentations and case studies on lessons learned.


The Uniqueness of Messenger RNA-based Therapeutic Products

Session Chair: Kathleen Francissen, Genentech, a Member of the Roche Group

Messenger RNA therapeutics have been in development for several years, and are used to introduce genetic information for a protein into a cell of interest without integrating into the genome. They include RNA-based cancer immunotherapies, prophylactic vaccines against infectious diseases, as well as so-called transcript or protein replacement therapy. This session will address mRNA product design, which can include individualized therapeutic products, and manufacturing considerations, such as ribonuclease-free conditions and scalability. In addition, this session will address the current regulatory environment for mRNA-based products, including expectations for control strategies.


Autologous Supply Chain

Session Chair: Bryan Silvey, Kite, a Gilead Company

With the advance of cell therapy products through the global regulatory process to availability on the commercial scale to patients, these early products begin with starting material from the patient and end with a cyro-preserved drug product being delivered for adminstration at the treatment site. This session will provide a view on the challenges and opportunities presented in the autologous cell therapy journey from manufacturer sourcing/container closure systems to logistics supplier technology required for delivery of a safe and effiacious product.


Analytical Characterization

Session Chair: Svetlana Bergelson, Biogen

Analytical characterization plays essential role in development of cell and gene therapy products, from guiding process development, determining storage conditions and assessing stability, to ultimately being used for release of the product for use in patients. The complexity of cell and gene therapy products is reflected in the wide range of methods used to assess product quality. Multiple challenges exist, from defining Critical Quality Attributes and limitations of relevant retain materials due to small batch sizes, to limited tool box of standard methods and reference materials that can be used to appropriately characterize these complex products. Even well-defined tests, such as compendial tests used for biologics, may not be directly applicable to cell and gene therapy products due to material requirements.  This session will focus on various aspects of analytical characterization for cell and gene therapy products, including development of new advanced methods to support comparability assessments, improve product understanding, and provide better tools for batch release and stability testing.


Regulatory Updates from Across the Globe

Session Chair: Michael Boyne, COUR Pharmaceutical Development Company, Inc.

This session will provide a high level regulatory discuss anchored by from representatives from multiple regulatory bodies. The panel discussion will be question based and focus on current trends and topics crucial to Cell & Gene Therapy Products. Areas a focus may include perspectives on accelerated development strategies, regulatory convergence/divergence, process validation and manufacturing challenges, reference standard qualification, analytical characterization, and recent regulatory guidance. We want to explore the initiatives the agencies have undertaken towards these topics, regulatory challenges and how they have may be overcome.


Strategy and Considerations for the Development of Potency Assays

Session Chair: Francis Poulin, Sanofi

Potency is a critical quality attribute whose analytical testing for cell and gene therapy (CGT) products presents a number of challenges not typically encountered with more established biopharmaceutics. For example, the biological activity of a CGT product is most often the sum of many biological activities. The measurement of potency can therefore be achieved via a matrix of orthogonal biological, biochemical and/or biophysical assays that need to be evaluated for correlation with functional/animal/clinical studies as product development progresses. Potency assays are also essential for product characterization and the elucidation of structure-function relationships. The first bioassay is usually not fully representative of the product’s mechanism of action and additional investigations are needed to link biological activity and clinical outcome. Ideally, a tiered approach to potency assay development will culminate with the implementation of surrogate assay(s) for routine testing that demonstrate acceptable reproducibility, robustness, shorter release timeline and regulatory approval. Challenges in the development of potency assays can be due to the technical limitations of in vitro systems or the absence of reference standards or suitable reagents. Autologous cellular products, produced as patient-specific lots, present additional constraints due to the limited amounts of material and the short time available for testing. During this session we will explore various facets of potency assay development strategies, and review approaches towards the establishment of robust potency assays for cell and gene therapy products.


Process Development, Validation and Continuous Process Verification

Session Chair: Cindy Riggins, Novartis Pharmaceutical Corporation

As more cell and gene therapy products move into late stage development, marketing approval and post-approval stages, the need for robust process development, validation and continuous process verification is necessary for a strong lifecycle management approach. These products are diverse and as such the approaches to development, validation and continuous process verification will likely be unique and require distinct strategies.

A strong lifecycle management approach will need to include an integrated team including representation from process development, manufacturing, analytical, microbiology, quality assurance and regulatory functions. While design of process validation and continuous process verification follows the principles outlined in FDA, EMA and ICH guidance’s, there are often new complexities and challenges that must be dealt with for cell and gene therapy products. These include challenges in obtaining or modeling patient-derived starting materials and specialized manufacturing equipment and facility design. Continuous process verification protocols will necessarily be highly product-specific, but incredibly important in order to ensure the validated process remains in a state of control over time. Overviews of the three areas with case studies and/or lessons learned will be presented. 


Late Stage Development and Commercialization

Session Chair: Anthony Lubiniecki, University of Maryland, Baltimore County

The good news is that the early clinical trials appear to show evidence of biological activity. The clinicians and the thought-and-opinion leaders are cautiously optimistic. Everyone around the table turns to the CMC development people and says, “When can you be ready to support pivotal trials and when can you be ready to support a launch?” These are the important questions which face those who succeed in early development. The needs of late development and commercialization are substantially different from those of early development. The focus is no longer just making the product available for small numbers of recipients, but rather for populations of substantial size. The need for quality, reliability, and consistency replaces the need for innovation and speed. Assay development needs to be completed. Scale of operations needs to be consistent with market size, not clinical trial size. The impact of process, analytical, site and product changes need to be most carefully thought through and be consistent with the rigor of available comparability assessments. Commercial cGMP standards apply to commercial products.

Speakers and discussants will be present to share their experiences and observations on making this key transition from early development to late development and commercial manufacturing stages. 


Critical Starting Materials

Session Chair: Andreas Kuhn, BioNTech RNA Pharmaceuticals GmbH

The quality of the raw materials used in biopharmaceutical manufacturing can have a substantial impact on the quality of the final product. This is especially of high importance for products with such complex manufacturing processes as for cell and gene therapies. In this session, critical raw materials for cell and gene therapy products will be discussed from different viewpoints, including the manufacturer's, user's, and regulator's. Topics will include sourcing strategies, specifications, and incoming testing.


Standards Development for Cell & Gene Therapy Products

Session Chair: Andrew Weiskopf, Biogen

The diversity of product types and the evolving nature of technologies within the realm of cell and gene therapies pose a number of unique questions for development of standards. For a patient-specific drug product such as gene-modified autologous cells, what constitutes a reference standard material? How do we make the best use of viral vector reference materials that are not specific to a product’s serotype or transgene? What do best practices look like for certain assays if product-specific factors impact their performance on a case-by-case basis? Can we leverage existing standards across pharmacopeia, regulatory guidance’s, and standards development organizations to achieve our goals? In such a constantly changing field, how can we ensure that standards are a means to accelerate development of these products and not a hindrance?

In this session, we will feature various facets of standards development for cell and gene therapy products. Speakers will present their perspectives on the progress to date and the challenges that lie ahead in the development and adoption of industry-wide standards for materials, assays, and manufacturing/quality practices.


Comparability through Development of Advanced Therapy Medicinal Products

Session Chair: Margarida Menezes Ferreira, INFARMED-National Authority of Medicines and Health Products, Portugal

The most frequent questions asked in scientific advice procedures are related to the suitability of the comparability program to support the introduction of changes in the quality of advanced therapy medicinal products. These changes are desirable to ensure better performing processes and products and they are necessary to establish appropriate analytical tools. Nevertheless, the level of flexibility acceptable in early development is progressively reduced from the non-clinical stage to the pivotal clinical use.  Data filiation demonstrating representativeness of the non-clinical product to support safety of the batches for initial clinical trials should expand to a full comparability exercise where a higher degree of sameness is expected. Comparability remains an important tool required to support changes after MA where the process and the product are expected to be well defined and appropriately controlled by quality specifications and characterisation tools. As such, preparing for comparability should be ideally initiated in the early phase of product development and it should aim to define an extended analytical and experimental program to minimize the need for additional non-clinical or even clinical data.

The present session presents challenging comparability issues raised with cell based as well as gene therapy products and the multitude of parameters to be considered as relevant for product integrity. Cell based products are very complex in terms of composition and dynamic nature, with the manufacturing process depending on the combination of multiple biologically active reagents, and often in unstable differentiation or tri-dimensional forms. Gene therapy products and specially the genetically modified cells are challenging products as they encompass critical aspects related to the gene technology as well as the complexities of the production based on patient cells. Manufacturing of the viral vectors and respective producer cell lines, vector optimisation, changes in cell packaging system or production scale, different intermediates and the patient cells to be transduced, changes in critical raw materials, multiple manufacturing units, multiple donors are all components to be considered.

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