Name: Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing...
Start: 2023-10-19T14:00:00Z
End: 2023-10-19T14:01:02.000Z
Location: BrightTALK

BioPharma Webinars aims to keep its global audience abreast of all developments in the areas of Drug Development, Manufacturing, Quality Assurance, Outsourcing and Regulatory Affairs, with only the highest quality webinars, presented by the most respected people, working with companies in Pharmaceutical, Biopharmaceutical, Biologics and Biotech Industry.

Full title: Regulatory Challenges of Recent Low Endotoxin Recovery Regulatory Queries: Pfizer’s Strategy to Evaluate and Implement Global Changes to Meet Evolving Expectations

The frequency and scope of inquiries related to Low Endotoxin Recovery (LER) from health authorities for biotherapeutic product submissions have dramatically increased over the past year. LER studies are required by the Center of Drug Evaluation and Research (CDER) – Food and Drug Administration (FDA). The Parenteral Drug association (PDA) Technical Report (TR) No. 82, Low Endotoxin Recovery, is used as a guidance for performing LER studies for Biologics License Application (BLA) submissions to CDER, although some recent queries diverge from PDA TR 82. In September 2023, the European Medicines Agency (EMA) published Questions and Answers on biological medicinal products, containing the question, “When should Low Endotoxin Recovery (LER, also known as “endotoxin masking) be investigated?”. 
The provided answer to this question does not specifically define LER or offer detailed
instructions on study methodologies. Due to the increase in queries, an assessment of the queries in a trackable database was completed. This presentation will highlight the major themes observed in these queries, particularly those that had tangible impacts on our LER strategy.

Regulatory Challenges of Recent Low Endotoxin Recovery Regulatory Queries

Full title: Addressing Subvisible Silicone Oil Droplets-Industry Challenges, Analytical Strategies, and USP's Rationale for a New General Informational Chapter

Subvisible silicone oil droplets (SiOPs) originating from siliconized prefilled syringes have become a significant contributor to subvisible particulate matter (SbVP) in biopharmaceutical products. While silicone oil is essential for device functionality, dispersed droplets complicate compendial particle measurements, challenge method specificity, and may interact with therapeutic proteins. This presentation synthesizes insights from a recent Pharmacopeial Forum Stimuli article titled and “Addressing Subvisible Silicone Oil Droplets—Industry Challenges, Analytical Strategies, and USP's Rationale for a New General Informational Chapter”.

We will examine the industry context of subvisible silicone oil droplets (SiOPs) across evolving delivery systems, including prefilled syringes and autoinjectors, and various administration routes except intravitreal use. This overview will compare analytical strategies such as light obscuration, flow imaging with morphology filters, orthogonal methods like NMR/ICP-MS and Raman/FTIR, and emerging tools such as holographic microscopy and AI-enabled image analysis, focusing on their limitations, variability, and fit-for-purpose application. Clinical and safety data will be discussed, emphasizing the low intrinsic toxicity of silicone oil and the context-dependent nature of immunogenicity risks. We will also outline development and lifecycle control strategies—covering siliconization processes, container selection, formulation factors, and manufacturing controls—and present a risk-based framework for product-specific specifications and regulatory engagement when SiOPs impact apparent SbVP levels.

Addressing Subvisible Silicone Oil Droplets-Industry Challenges, Analytical Strategies, and USP's Rationale...

This presentation explores the use of solid phase cytometry for in process bioburden, short shelf-life product and water testing. Solid phase cytometry is being looked at as the new frontier for pharmaceutical microbiology especially for cell and gene therapy. The presentation looks at the practicality, use and limit of detection of these methods in a GMP QC environment and determines what further work might be required. It focuses mainly on low bioburden and in process testing for small molecules. Other new technologies are discussed such as solid phase real time imaging and real time imaging for pharmaceutical development.

Sophie Drinkwater, Associate Director in Microbiology at AstraZeneca

The development of mRNA-based medicines requires robust potency assays to assess both translatability and biological activity. These assays can be either cell-free or cell-based assays; each provide a different assessment but are complementary to each other. As such, we will discuss various strategies for assessing mRNA DS and DP potency evaluation and the advantages and disadvantages between cell-free and cell-based assays.

mRNA Products: An Overview on Strategy for Potency for DS vs DP

Medium-chain fatty acids are used extensively as additives to plastics and elastomers. Plastics and elastomers are in turn used in the fabrication of components which then are used in the fabrication of drug product container closure-delivery systems and medical devices. As a result, these medium-chain fatty acids routinely appear in extractables profiles from these components as well as in leachables profiles of drug products. The most commonly used medium-chain fatty acid additive is “Technical Grade Stearic Acid” either as the free base or salt (e.g., Mg, Ca, Zn). Technical Grade Stearic Acid contains a mixture of fatty acids, with Palmitic (C16) and Stearic (C18) acids being the most abundant. These fatty acid mixtures are of biological origin, either plant or animal derived (bovine). It is well established in the scientific literature that the ratios of C16/C18 in fatty acid mixtures vary with origin. 
This presentation focuses on the use of extractables and leachables profiles to indicate the origins of fatty acid additives in packaging and medical device components. Data will be presented from both GC/MS and LC/MS analyses of various extractables/leachables profiles and compared with the chromatographic profiles of fatty acid mixtures of known origin. It will be shown that plant and animal derived C16/C18 ratios can be distinguished by either analytical technique, alerting the analytical chemist to the possibility of a bovine sourced additive with the potential of carrying Bovine Spongiform Encephalopathy (BSE).

Fatty Acid Extractables/Leachables Profiles as an Indicator of Technical Grade Stearate Acid Additive Origin

Over the past few years, the mass spectrometry-based Multi-Attribute Method (MAM) has gained traction due to its potential to streamline lab work and decrease development time. However, several barriers to adoption remain, one of which is evaluating performance of MAM vs. conventional methods.  

To evaluate the performance of MAM vs conventional methods, adalimumab and etanercept were procured from multiple sources, and subjected to forced degradation to generate a range of modifications. Samples were analyzed using both conventional and MAM approaches to evaluate size variants, charge variants, glycosylation, sialic acid content, and other molecular variants like oxidation, deamidation, and glycation.

The ability of each technique to detect differences between nominally similar products and forced degradation-induced changes was evaluated. Physiochemical differences were compared to functional and structural changes detected using cell-based assays, binding assays, and circular dichroism. The knowledge base from this study can help lower the barrier to adoption of MAM, enabling greater efficiency and reducing costs.

Enabling Broader Adoption of MAM: Comparison of MAM vs. Conventional Methods

The absence of adventitious agents is critical for the quality of pharmaceuticals, where contamination event can arise from starting materials or during production (e.g., cell line, cell substrates, equipment, facilities and/or operators). 
 
Rapid microbial methods offer alternatives to traditional compendial methods: reduced time for testing, reduced sample volume requirement, possible automation, increased sensitivity and accuracy. These technologies are most suitable for products with a short life, limited supply, and urgent needs and which may be infused into patients before the completion of the test.
 
The presentation will provide a general overview of the official USP RMM chapters and those under drafting or commenting. In addition, the frequently asked questions, general principles and future developments of the USP <72> and <73> chapters will be explained in detail.

Overview and Future Plans of the USP Rapid Microbiological Methods Subcommittee

Advances in extreme ultraviolet lithography have unlocked the computational power needed to produce chips and GPUs capable of training and deploying GenAI models that are reshaping the way we work and interact with technology. These breakthroughs have been both frightening and inspiring for the manufacturing industry. The ability of these models to process massive amounts of data and deliver insights in ways humans can easily interpret is truly remarkable.

Yet, because AI has not been part of foundational education curricula, widespread misunderstandings persist—even among technically skilled professionals outside the AI field. Market pressures to include “AI” in products and business strategies are also creating significant pressure on executive teams to invest in AI applications without a clear understanding of potential use cases, implementation requirements, or expected return on investment.

In this webinar, I will explore key considerations for integrating various forms of AI—not just GenAI—across the pharmaceutical value chain, with a particular focus on manufacturing and quality. My goal is to provide executives and managers with actionable insights to define realistic strategic objectives, achieve measurable ROI, and avoid common pitfalls in AI adoption.

Developing and Deploying Digital & AI Solutions in Pharma: The Reality vs. the Hype

The emergence of mRNA modality as a therapeutic alternative for treatment of viruses, infectious disease, and various therapies has highlighted the need for high quality mRNA. To meet the quality requirement, developers continue to examine raw material quality, process controls and appropriate analytical methods to ensure the manufacturability of high quality mRNA. As such, we will discuss various strategies to ensure the manufacturability and characterization of high-quality mRNA.

Strategies for Controlling the Quality of mRNA

Detection of bacterial endotoxins is a critical quality attribute of parenteral pharmaceuticals and medical devices.  Bacterial endotoxins are cell wall components of Gram-negative bacteria, ubiquitous in nature, difficult to destroy and can cause pyrogenic responses or worse.

We are at a generational change in bacterial endotoxins test method options.  Creating the infrastructure to enable broader use of recombinant reagents advances the science while maintaining the safety and quality of medicines.  Recombinant reagents are derived from established biotechnology principles which begin with the same mechanism of action as the natural protein derived from horseshoe crabs; the detection of bacterial endotoxins by the Factor C protein.  Recombinant reagents also afford improvements in quality, supply chain, and sustainability.

The USP Microbiology Endotoxin and Pyrogens Subcommittee developed a recombinant reagent bacterial endotoxins test chapter which is based on the underlying science, and data from the peer-reviewed literature, sponsor and supplier validations, and ring study trials.

The presentation will provide a general overview of the Subcommittee work, and detailed information regarding the recombinant bacterial endotoxins chapter, associated guidance, and the public health reference standard endotoxin.

An Overview of Recent USP Bacterial Endotoxins Standards Development

The Pfizer Continuous Manufacturing Module (PCMM) is capable of manufacturing tablets via either direct compression (DC) or wet granulation (WG).  The initial commercialization of DaurismoTM (Glasdegib) and CibinqoTM (Abrocitinib) was achieved in 2018 and 2021 respectively using a PCMM- DC continuous direct compression approach. In this presentation we will discuss the development and application of PCMM-WG technology to Pfizer’s third NCE small molecule compound.  We will discuss our rational for drug product design, components of the emerging control strategy, and an overview of multi variate experimental and modeling efforts supporting this effort.

Expanding Continuous Manufacturing at Pfizer, Enabling High Drug Load Formulations via Wet Granulation Technology

Conjugate vaccine development is a complex, complicated and lengthy process comprising of multiple antigenic vaccine components. The Serum Institute of India, a global leader, employs advanced data analytics and Design of Experiments (DoE) principles to develop and optimize Vaccines R&D and manufacturing. By integrating tools like Advanced Data Analytics, we've significantly enhanced yield consistency and process robustness from the initial stage of development.

This presentation will explore how advanced analytics and smart manufacturing are revolutionizing our vaccine development and production approach. We'll discuss addressing complex scientific challenges, scaling up early-stage data for commercial manufacturing, and leveraging real-time data analytics through high-performance online sensors.

I will also share insights into the application of Online Data Analytics platforms in establishing flexible QbD frameworks. Join us to discover how data-driven decision-making is driving impactful advancements in vaccine manufacturing using Predictive & Prescriptive analytics.

Vaccine Development using DoE and its Manufacturing Technology with the application of Advanced Data Analytics

While control strategies, including Process Analytical Technology (PAT) and Real-Time Release Testing (RTRT) elements, are common in oral solid dosage forms (with in-process testing and spectroscopic methods widely accepted by health authorities), their application in aseptic manufacturing remains rare. This webinar details a pioneering use case at Roche: the successful integration and regulatory filing of RTRT elements into the release control system of a sterile biotech drug product at a commercial launch site.

Key components of this RTRT control strategy included:

<li> Implementing a dedicated RTRT sampling point.

</li><li> Utilizing Raman spectroscopy as a multi-attribute method.

</li><li> Leveraging the existing rapid microbiology toolbox.

The presentation will also address regulatory feedback and lessons learned during this implementation</li>

Crossing the Finishing Line: The first Implementation of RTRT/PAT for a BioTech Drug Product at Roche

Genetic characterization of mammalian cell lines prior to registrational filing is a regulatory requirement documented for example in ICH Q5D. It is required to demonstrate that a cell line is genetically stable throughout the manufacturing process to ensure manufacture of a consistent product.

Several different approaches for genetic characterization of cell lines are common in the industry.  Firms perform assays internally, or outsource testing to Contract Testing Laboratories. External testing is mostly performed using GMP test methods; for internal testing, there is about an equal split between use of GMP and non-GMP methods.

In this presentation, I’d like to challenge the need of GMP test methods for genetic characterization  of cell lines.

Challenging Standards for Genetic Characterization of Manufacturing Cell Lines

Full Title: Potency Assays and Functional Characterisation—Going Hand-in-Hand during Product Development up to Commercial Licensing

Presented by Therese Choquette, PhD. Head of Analytical and Translational Sciences at Tigen Pharma, Ulrike Herbrand, Scientific Director Global in vitro Bioassays and Head of the Bioassay Research & Development team at Charles River Laboratories and Felix Montero Julian, Vice President of Scientific Affairs at Accellix

The potency assay is a highly critical assay for cell and gene therapy products and is one of the assays on the release specification. The potency assay needs to reflect the main mechanism of action/s of the product and the CQA. Using cellular function characterisation assays during development provides critical insights which help to establish the most relevant and appropriate potency assay for the product.

Adressing the CQA Potency for ATMPs: A Risk-Based Phase-Appropriate Approach

Determining which attributes are critical to the quality of the product and the safety of the patient is individual to each product. During the lifecycle of an ATMP managing CQAs evolves through the different phases of development (early phases might focus on identifying potential CQAs and understanding their impact, while later phases involve more rigorous control and validation). Potency assays are a major challenge since often the mechanism of action (MoA) of the product is not fully understood in early phases. In those cases, a matrix approach with incremental development using less complex methods in the beginning can help to ensure avoiding delays on the path to market. Alignment with regulatory guidelines is crucial at any stage.

Potency Assays and Functional Characterisation

Ensuring viral safety in oncolytic virus manufacturing can be particularly complex when neutralizing antibodies interfere with traditional biosafety assays.

In this session, we will explore how GMP NGS enables accurate detection of adventitious agents and supports risk mitigation when antibodies threaten viral safety assessment. You’ll gain insight into regulatory expectations, case studies, and how integrating NGS-based biosafety into your workflow can de-risk development and accelerate timelines.

Key takeaways:

Addressing antibody neutralization in oncolytic virus GMP manufacturing
Enhancing biosafety with validated NGS approaches
Real-world applications and regulatory insights

Secure your place today and explore how GMP NGS empowers oncolytic virus manufacturers to ensure viral safety and, speed up timelines.

Oncolytic Virus GMP Manufacturing: Solving Antibody Neutralization Challenges with Next Generation Sequencing

Join Scott Mangiacotti, Senior Director, Automation Solutions at Cytiva, for a webinar on using pre-engineered, process-ready solutions to transform how automation is deployed and scaled across your operations. Compatible with leading industrial automation platforms, these pre-engineered solutions are built on open standards and are digitally enabled to support cloud connectivity, advanced process control (APC), and process analytical technologies (PAT).

We’ll explore how productized, scalable designs can help you reduce project timelines, lower total cost of ownership, and minimize implementation risks—all while maintaining performance and flexibility. Whether you're planning a new automation project or enhancing existing infrastructure, this session will provide practical strategies to save time, reduce costs, and drive smarter industrial operations.

Drive Efficiency With Pre-engineered Solutions for Industrial Automation

The quality and purity of mRNA are critical for vaccine and therapeutic safety. As new technologies emerge, bridging analytical methods, especially in impurity detection, remains challenging. Comprehensive characterization is essential for identifying impurities like dsRNA and residual DNA. USP is developing documentary and physical standards which support the mRNA product lifecycle. This presentation explores evolving mRNA analytics and opportunities to enhance impurity detection and improve product reliability

Navigating mRNA Impurities: A Focus on Quality

Full title: Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing Demand For these Viral Vector Delivery Systems

Presented by Seth Levy, Director of Bioprocess Development at Modalis Therapeutics and Gael Nicolas, Senior Technical Sales Specialist at Refeyn

Modalis’ epigenetic editors are designed to efficiently target genes and control chromatin conformation by coupling an engineered Cas9 variant with epigenetic effector domains; referred to as GNDM (Guide Nucleotide Directed Modulation), which is delivered via a single AAV vector.Most AAV capsids used for systemic treatments require high doses, leading to safety concerns. To overcome such obstacles, Modalis has evaluated numerous novel AAV capsids allowing for reduced dosing, increased safety margins, and higher therapeutic efficacy. These novel capsids do not always play nicely with standard manufacturing techniques and available reagents, so creative process development and analytics are needed to bring these promising therapeutic molecules to patients.

Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing...

Viral Vectors

Viral Vector Delivery Systems

Mass Spectrometry

Biologics

Biotech

Epegentic Editors

AAV Capsids

Therapeutic Efficacy

Therapeutic Molecules

Gene Therapy

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