Conference: Bioassays/Potency Assays – Regulatory Requirements, Development and Routine Use

Objectives

The newly developed bioassay/potency assay conference will cover both existing and a number of developing regulatory guidelines and representatives from three different European and US regulatory authorities will talk about relevant guidelines, monographs and their expectations and experiences. In addition, experts from industry, contract research organisations and CDMOs as well as scientists from fields such as cell banking, mathematics/statistics and more will present their work in the development, validation and routine use of potency assays. In addition to various modalities from classical proteins to ATMPs and vaccines, experts will provide insights into the automation of procedures and the use of AI.  The conference is intended to provide a platform for scientists, experts and representatives of authorities to exchange knowledge and opinions and to discuss experiences and expectations.

Background

The number of biopharmaceutical products in the clinic and on the market continues to grow. The focus is increasingly on the area of cell and gene therapeutics or ATMPs. Many of these products are characterised by a high level of complexity. As a result, their bioactivity cannot be measured using conventional analytical tests alone. In addition to the special requirements, e.g. for product and process-related factors such as difficult upscaling, handling and testing of small and very small batches, quality of the starting materials and limited shelf life, the development of suitable potency assays is often a challenge. This includes the fact that one or more assays are usually required for each product, that there are no reference standards and that the leap from development to the GMP-regulated area is not always easy. At the same time, however, bioactivity is an indispensable CQA for release analyses and must be addressed. The MoA is also not always easy to determine, especially with CGT. The FDA also says in its guide: "However, many CGT products have complex (e.g., rely on multiple biological activities) and/or not fully characterized mechanisms of action (MOA), making it difficult to determine which product attributes are most relevant to measuring potency. A further factor is that the regulatory background for these new products is only gradually developing and the authorisation and supervisory authorities and pharmacopoeias have only recently published or still have to draft corresponding guidelines and monographs." A further factor is that the regulatory background for these new products is only gradually developing and the authorisation and supervisory authorities and pharmacopoeias have only recently published or still have to draft corresponding guidelines and monographs.

 

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Target Audience

• Representatives of the regulatory and authorisation authorities
• Specialists for biopharmaceutical manufacturing processes
• QA/QC personnel in the biopharmaceutical environment
• Laboratory staff involved in the development and routine use of bioassays/potency assays
• Project managers and outsourcing personnel
• Biologists, analytical chemists and biochemists
• Scientists from academic fields involved in the development of biopharmaceutical products

Moderation

Dr Alexander Knorre, Eurofins

Tuesday, 26 November 2024

Key Note on 26 November: The Promise and Challenges of In Vitro and In Silico Models in Drug Development
Dr Julia Schüler, Charles River Laboratories
The presentation will highlight important developments in the drug development technology landscape influenced by the concept of 3R and the evolving legal landscape. General characteristics of the different applications, their translational relevance as well as adoption drivers will be discussed. Case studies from oncology drug development will help to elucidate these trends and their impact on future processes.

Potency Testing Approaches: Challenges and Opportunities for mRNA Therapeutics
Dr Jan Michel Falcke, BioNTech

• Biological activity of mRNA products is a complex interplay between the mRNA drug substance properties and the effective delivery system to target cells
• Provide insight into the mechanism of action for mRNA products with a focus on different analytical techniques and a respective analysis of their advantages, disadvantages and key challenges
• Share industry perspective on crucial aspects regarding quality control measures and regulatory considerations on the application of potency assays as part of the release testing of commercial mRNA products
• Explore the testing of multi-construct products, adding depth to our understanding of the complex landscape surrounding mRNA product development

Challenges with Bioassay Design for mRNA Therapeutics
Thomas Ludwig, VelaLabs

• What’s the principle of a relative potency assay?
• Cell-based potency assay development and its challenges
• Qualification according to ICH Q2(R2) and defining reasonable suitability criteria for routine analytic
• Observations when measuring stability study samples

A Versatile Multiplexing Technology for Complex Drugs Characterization and Potency
Dr Rosaria Esposito, bioMérieux

• Considering the challenges with the emergence of new vaccine platforms and vaccines becoming more and more multivalent, current analytics methods are no longer adapted
• The potency testing for mRNA vaccines, such as the protein expression, is complicated due to the protein’s low concentrations especially in multivalent formulations
• Studying the immune response is challenging for highly multivalent vaccines, like pneumococcal. It requires complex testing plans and contributes to increase the time to market
• We will present a protocol and data for:
  • The highly sensitive detection of expressed proteins in a multiplex cell-based potency assay
  • Multiplex serological sample testing

Enhancing Bioassay Precision and Throughput with Modular Workflow Automation
Dr Sean Lin, Eurofins

• Challenges of automation within the confines of a QC release testing environment compliant with Good Manufacturing Practice (GMP) standards
• Implementation of semi-automation in various bioassays for modular workflow automation
• Improving reliability, precision and throughput of our bioassays

Harnessing the Power of Automation for Potency Assays and for Large-Scale Potency Assay Cell Bank Production
Sheri Mahan-Hunter, Pfizer

• Automation of Potency Assays
• Transfer of automated Potency Assays to GMP Laboratories
• Introduction of Frozen Ready to Use Cells (FRTU)
• Large-Scale Cell Banking Using Automation

Advancing Potency Assay Automation
Dr Katharina Künzel, Boehringer Ingelheim

• Challenges of potency assay automation
• Automation in development and routine

Potency Assurance for Cellular and Gene Therapy Products
Dr Andrew Byrnes, FDA/CBER

• An overview of FDA’s draft guidance on potency assurance for cellular and gene therapy products
• Advice on potency assays
• Other aspects of potency

Potency Assays as Part of Release Testing for ATMPs – Focus on AAV
Dr Christoph Mück, AGES

• How is potency defined?
• Requirements for potency assays
• Potency assays for adeno-associated virus ATMPs
• Challenges in assay development

Development of Methods for Comparative Analysis of the Potency of Monoclonal Antibodies
Dr Lilija Miller, Paul-Ehrlich Institut
Growing number of monoclonal antibodies leads to a large number of potency assays

• In case of e.g. counterfeiting or theft of monoclonal antibodies, the Paul-Ehrlich-Institut (and also other authorities) cannot perform ad hoc potency testing due to lack of expertise/experience and the large number of possible potency assays
• In a BMG-funded project, comparative analyses of the potency of monoclonal antibodies are to be established and thus ideally reduce the number of potency assays to be kept in stock