CASSS Welcome and CMC Strategy Forum 2024 Introductory Comments

Jan 22, 2024 8:30am ‐ Jan 22, 2024 8:50am

Health care providers and patients are frequently required to manipulate biological drugs prior and during administration. This might include procedures such as dilution of the drug into admixtures for infusion, limited storage, transportation to the hospital or exposure to new contact materials, such as intravenous bags.

The instructions for use are provided by the drug manufacturer and are based on studies to support in-use stability and compatibility with administration components, simulating drug handling and hold times throughout the defined in-use period. The studies are challenging, because there is a wide range of in-use conditions and administration components used globally. In addition, only limited guidance is available from regulators on expected in-use stability data.

In four workshops we will present and discuss industry approaches to assess in-use stability and compatibility for classic biological products as well as emerging trends. Each workshop is scheduled for 90 minutes, and includes 2 presentations followed by a discussion with the presenters and additional panel members.

CASSS Welcome and CMC Strategy Forum 2024 Introductory Comments

Jan 22, 2024 8:30am ‐ Jan 22, 2024 8:50am

Platform technologies are used extensively across the biopharmaceutical industry to develop drugs efficiently and effectively.  The application of prior knowledge and platforms from established products lays a strong foundation to guide development and advance new medicines to the patients. From cell line development, upstream and downstream operations, formulation development, devices, and analytical methodologies, platforms have proven that they work and are a powerful tool.  However, there are no universal standards on how to create, justify and maintain platforms. The inclusion of platform information and the level of detail in the dossier is key for regulatory agencies to evaluate the applicability of the platform using a risk-based assessment.  Moreover, the rationale and justification on how prior knowledge and platform data are suitable for the intended product is essential in a review.  This session will look at the application of platform approaches and technologies and how they can be applied to programs to support development and registration. Insight into how prior knowledge for a platform is fully leverage across products and supporting rationale will be presented in case studies.  The objective from the session should help developers and health authorities better align on expectations and paths to use platforms for clinical development through commercialization.

Workshop I: Analytical

Jan 22, 2024 8:50am ‐ Jan 22, 2024 10:20am

Session Speakers:

Platform Technologies Designation Program: Highlights of Statutory Language

Phillip Kurs, CBER, FDA

Leveraging Prior Knowledge for Practical Application of Platform Analytical Procedures in Late-Stage Development of Monoclonal Antibodies

Hetalben Patel, Pfizer, Inc.

Platform Approaches in Analytics for rAAV based Gene Therapies 

Van HoangSpark Therapeutics, Inc.

Planning Platform Analytical Approaches for Cell Therapy

Emily English, Cartesian Therapeutics, Inc.

Workshop I: Established Best Practices for In-Use Stability Study Design and Testing

Jan 22, 2024 8:50am ‐ Jan 22, 2024 10:20am

Session Speakers:

How to Design and Perform In-Use Stability and Compatibility Studies?

Markus Blümel, Novartis Pharma AG


In-Use Stability Study Design: A Regulatory Perspective

Paula Russell, Health Canada 

Additional Panelist:

Ankit Patel, Denali Therapeutics 

Isabella de Jong, Genentech, a Member of the Roche Group

Workshop I: Panel Discussion: Analytical

Jan 22, 2024 10:50am ‐ Jan 22, 2024 12:20pm


Emily English, Cartesian Therapeutics, Inc.

Phillip Kurs, CBER, FDA

Van Hoang, Spark Therapeutics, Inc. 

Hetalben Patel, Pfizer, Inc., 

Da Ren, BioTherapeutics Solutions, Inc.

Workshop II: Microbial In-Use Studies and Closed System Transfer Devices

Jan 22, 2024 10:50am ‐ Jan 22, 2024 12:20pm

Session Speakers:

In-Use Studies Microbial Challenge Studies: Cross-Industry Efforts to Harmonize Strategies in Collaboration with Health Authorities

J. Paul Kirwan, Amgen Inc. 

Pharmaceutical Industry Perspective on Closed System Transfer Devices (CSTDs): Balancing Overfill, Evaluating, Communicating

Christian Lehermayr, Novartis Pharma AG

Additional Panelists:

Virginia Carroll, CDER, FDA

Nicholas Clark, Amgen Inc.

John Metcalfe, CDER, FDA

Workshop II: Process

Jan 22, 2024 1:20pm ‐ Jan 22, 2024 2:50pm

Session Speakers:

Incorporation of Platform Based Approaches and Prior Knowledge Leveraging Into the Recently Revised ICH Q5A (R2) Guideline

Kathryn KingCDER, FDA

Power of Platform to Accelerate Therapies Beyond a Pandemic 

Ronan Kelly, Eli Lilly and Company 

Leveraging Platforms to Accelerate ADC Formulation and Drug Product Development

Karen Rutherford, Pfizer Inc.

Continuous Platform Improvement to Propel Drug Substance Development,

Heather Nunn, Amgen Inc. 

Workshop III: Product In-Use: Meeting Patient and User Needs

Jan 22, 2024 1:20pm ‐ Jan 22, 2024 2:50pm

Session Speakers:

Patient and User-Centric Considerations on In-use Study Design

Sarah Weiser, Pfizer, Inc

Leveraging AI to Meet Patient Needs: Improving Product Quality in Biotechnology

Prashanth Chodagiri, Amgen Inc.

Additonal Panelist:

Sarah Donegan, AstraZeneca 

Workshop II: Process - Panel Discussion & Closing Remarks

Jan 22, 2024 3:15pm ‐ Jan 22, 2024 5:00pm


Carl Frye & Ronan Kelly, Eli Lilly and Company

Kathryn King, CDER, FDA

Heather Nunn, Amgen Inc.

Karen Rutherford, Pfizer Inc.

Dean Smith, Health Canada

Workshop IV: Emerging Trends in Compatibility and In-Use Stability Studies & Closing Remarks

Jan 22, 2024 3:15pm ‐ Jan 22, 2024 5:00pm

Session Speakers:

In-Use Compatibility Testing of Cell and Gene Therapies (CGT)

Philip Grossen, F. Hoffmann-La Roche Ltd.

Abbygail Foster, Genentech, a Member of the Roche Group

Challenges of In-Use of Low – Dose High Potent Product- Case Studies and Potential Mitigations

Basma Ibrahim, AbbVie Inc.

Additional Panelists:

Andrew Chang, Novo Nordisk Inc.

Martin Nemec, Health Canada

Jennifer Swisher, CDER, FDA

CASSS Welcome, WCBP Introduction & 12th Annual William Hancock Award

Jan 23, 2024 8:00am ‐ Jan 23, 2024 8:45am

Keynote Presentation: Disruptive Innovation in Drug Development and Personalized Medicine: Human Organ Chips and Beyond

Jan 23, 2024 8:45am ‐ Jan 23, 2024 10:00am


Donald E. Ingber, Wyss Institute for Biologically Inspired Engineering, Harvard University 

The large number of failures observed in human clinical trials is a problem that must be overcome for the benefit of patients and the survival of the pharmaceutical industry. Failure of animal models to predict therapeutic responses in humans is a major part of the problem.  In this presentation, I will describe Organ-on-a-chip (Organ Chip) microfluidic devices lined with living human tissues that form tissue-tissue interfaces, reconstitute vascular perfusion and organotypic mechanical cues, integrate immune cells, contain living microbiome, and recapitulate organ-level physiology and pathophysiology with high fidelity. Work will be presented describing how single human Organ Chips and multi-organ human Body-on-Chips systems have been used to model complex diseases and rare genetic disorders, study host-microbiome interactions, quantitatively predict drug pharmacokinetic and pharmacodynamic parameters, recapitulate whole body inter-organ physiology, and reproduce human clinical responses to drugs, radiation, toxins, and infectious pathogens. We also have used human Organ Chips to gain new insight into mechanisms of host immunity to viral infections and to develop new therapeutics for potential pandemic respiratory viruses, including influenza and SARS-CoV-2. My message is that the possibility that human Organ Chips can be used in lieu of animal models for drug development and as living avatars for personalized medicine is coming ever closer to becoming a reality. In addition, I will briefly review new technologies being pursued at the Wyss Institute at Harvard that I lead, including artificial intelligence approaches for accelerated drug discovery, novel drug shuttles that cross the blood-brain barrier with high efficiency, and handheld multiplexed companion diagnostic devices for clinical trials and home healthcare.

Plenary Session 1 - Novel Modalities

Jan 23, 2024 10:30am ‐ Jan 23, 2024 12:00pm

Technological innovation and advancements have enabled novel designs for antibody-based products, vaccines, blood products, gene and cell therapy candidates. While mAB conjugates like ADCs and novel mAB constructs like bispecifics molecules have been maturing as approved therapies for unmet medical needs, additional molecules like mAb fragments, trispecifics, cocktails, bifunctional (e.g., mAb-cytokine fusion proteins), and non-IgG isotype molecules are in development. Novel approaches to vaccine design include the expression of antigen to elicit a protective immune response and development of universal vaccines for flu and SARS-CoV-2. Gene and cell therapies continue to evolve, including new designs for CAR-T cell constructs to decrease side effects, improve proliferation and cytotoxicity, and increase specificity of therapeutic targets.  The emerging allogenic/ Induced Pluripotent Stem Cells (IPSC) CAR-T cells will further the reach of CAR-T applications.  In addition, macrophage CAR which are in development may better target solid tumors. This springs the questions, do novel modalities warrant novel product quality controls because of product quality concerns? It is acknowledged that based on the modality, additional testing based on novel analytical techniques, control and characterization may be recommended and should be determined on an individual case basis. This should be informed by scientific knowledge and risk-based approach aligned with guidance documents. In this session we will discuss the industry’s current trend and application of innovative approaches for new modalities, with a focus on the success, challenges, and lessons learned during product development. 

Session Speakers:

Enhanced Quality Attribute Understanding Enabled Accelerated Development of a RSV Vaccine

John Davis, Pfizer, Inc.

Analytical Considerations in the Development of Engineered Therapeutic IgM Antibodies

Devinder Ubhi, IGM Biosciences, Inc.

Jaime Marach, IGM Biosciences, Inc.

6+ Years of Autologous CAR-T Therapy  

Mehrshid Alai-Safar, Kite, a Gilead Company

From High Order Aggregates to Peptide Maps: Comprehensive Protein Characterization Using Advanced UHPLC-HRAM MS Platforms | Presented by Thermo Fisher Scientific

Jan 23, 2024 12:30pm ‐ Jan 23, 2024 1:30pm

Session Speakers:

From High Order Aggregates to Peptide Maps: Comprehensive Protein Characterization Using Advanced UHPLC-HRAM MS Platforms

Andrew Mahan, Janssen Research & Development, LLC

Welcome Day 2 & CASSS Distinguished Fellows Awards

Jan 24, 2024 8:15am ‐ Jan 24, 2024 8:30am

The designation of CASSS Distinguished Fellow is designed to recognize individuals noted for their outstanding contributions to CASSS, our community and the industry overall.  These individuals are honored with the title of Distinguished Fellow, bestowed on less than 1% of our members, and offered lifetime membership in CASSS. Join us this morning as we recognize two deserving individuals as new CASSS Distinguished Fellows.

Plenary Session 3 - State of the Art, Science-Based Comparability Strategies and Novel/Platform Technologies

Jan 24, 2024 8:30am ‐ Jan 24, 2024 10:00am

Throughout the life cycle of a biotherapeutic it is often necessary to make changes to the manufacturing process and control system. These changes may improve the consistency of the process, increase the scale or yield to meet supply needs, involve improvements to analytical methods, transfer to a new facility or equipment, and/or be designed to improve product quality. When these changes occur it is necessary to demonstrate that product quality is not adversely impacted, and that the safety and efficacy established with prior process versions will be maintained with material from the post-change process. This plenary session will focus on state of the art, science-based and regulatory strategies that enable process changes to be executed successfully and efficiently. In scope topics may include comprehensive comparability strategies guided by patient-centric considerations, lean technical transfers enabled by the use of platform technologies, and perspectives on the use of regulatory tools to support changes.

Session Speakers:

Building a Comparability Strategy for a Major Post-Approval Manufacturing Change

Nathan McKnight, Genentech, a Member of the Roche Group

An Industry Perspective on the Use of Forced Degradation Studies to Assess Comparability of Biopharmaceuticals

Kasia Nowinski, Pfizer, Inc.

Accelerating Biopharmaceutical Development: Data-Driven Strategies, Platforms, and Technologies

Nitin Rathore, Amgen Inc.

Additional Panelist:

Leslie Rivera Rosado, CDER, FDA

Host Cell Protein ELISAs: The Scientific and Business Decisions to Ensure Successful Outcomes | Presented by Cygnus Technologies, LLC

Jan 24, 2024 12:15pm ‐ Jan 24, 2024 1:15pm

Session Speaker:

Host Cell Protein ELISAs: The Scientific and Business Decisions to Ensure Successful Outcomes

Eric Bishop, Cygnus Technologies, LLC

Potency Assays for ATMPs – Selection of Assay Platforms and Data Processing Methods are Key Aspects for Successful Control Strategies | Presented by BioAgilytix Labs, LLC

Jan 24, 2024 12:15pm ‐ Jan 24, 2024 1:15pm

Session Speakers:

Potency Assays for ATMPs – Selection of Assay Platforms and Data Processing Methods are Key Aspects for Successful Control Strategies

Jeff Patrick, BioAgilytix Labs, LLC

Shiqian Zhu, BioAgilytix Labs, LLC

Plenary Session 4 - Sustainable Biopharmaceutical and Vaccine Development – Strategies for Reducing Waste, Protecting the Environment, and Reaching Net-Zero

Jan 24, 2024 4:45pm ‐ Jan 24, 2024 6:15pm

Biopharmaceutical and vaccine development contribute significantly to global carbon emissions, plastic and chemical waste, water utilization, and animal testing.  Strategies to implement sustainable practices can have a large positive impact on our environment, while still maintaining efficient development of critical and lifesaving drugs and vaccines for the benefit of society.  New ways to streamline processes, optimize supply chains for raw materials and products, reduce testing on animals, substitute materials used in manufacturing and analytical assays, and to reduce solvent volumes through continuous manufacturing and real-time inline release testing can all be viable approaches for sustainable development. This session will focus on strategies and opportunities to incorporate reductions in resource utilization, energy, water and environmental impacts for more sustainable biopharmaceutical and vaccine development to reach net-zero carbon emission impact as an organization and then ultimately an industry. 

Session Speakers:

Considerations for Animal Use and Sustainability – A Regulatory Perspective

Robin Levis, CBER, FDA

Designing and Manufacturing Medicines & Vaccines with Sustainability at the Core
Phil Dell'orco, GlaxoSmithKline 

Enabling Sustainable and Predictable Bioprocessing Through Small-Footprint Automated Microbial Production
Chris LoveMassachusetts Institute of Technology

Plenary Session 5 - Drug Delivery Innovations for the Next Generation of Biologics and Vaccine Products: Smart Design, Smart Device

Jan 25, 2024 9:00am ‐ Jan 25, 2024 10:30am

The industry is committed to improving patient outcomes through the continuous development of innovative drugs, vaccines, and therapies. One area of focus for this innovation is drug delivery. 

Administration of biological drugs and vaccines is challenged by painful methods using needles or invasive methods, often adversely affecting patient compliance. For CNS drugs, currently the most common method of delivery is through invasive intra-CNS devices. Additionally, efficacy of parenteral drugs and vaccines can be negatively affected by non-optimal targeting to sites other than that of the natural biological pathway (e.g., injecting intramuscularly, intravenously, or subcutaneously when the site of the target is mucosal or in the CNS space).

This session highlights innovations intended to enhance the effectiveness, uptake and compliance of injectable biological drugs. Topics include smart molecular designs for less frequent administration, novel technologies for less painful delivery (e.g., microneedles), more effective routes of administration (e.g., intra-nasal), and smart devices with feedback mechanisms to optimize dose and frequency.

The desired outcome for this session is to feature the latest trends in drug delivery and understand how far the challenges can be overcome towards improving patient outcomes and advancing the field.

Session Speakers:

From Blood-Brain Barrier to Blood-brain Interface: Strategies for Drug Delivery to the Brain

Bill Banks, University of Washington

Trends and Developments in Biopharmaceutical Delivery Innovations

Tine Zachariasen, Novo Nordisk A/S

Drug Delivery Innovations for CEPI’s 100 Days Mission

Dimki Patel, CEPI

Characterization of Complex cIEF Electropherograms From mAb and Antibody-Drug Conjugate (ADC) Using a Novel icIEF-UV/MS System | Presented by SCIEX

Jan 25, 2024 12:30pm ‐ Jan 25, 2024 1:30pm

Session Speaker:

Characterization of Complex cIEF Electropherograms From mAb and Antibody-Drug Conjugate (ADC) Using a Novel icIEF-UV/MS System

Mingjie Cui, AstraZeneca

Untangling the Complexity Within Oligonucleotide Therapeutics | Presented by Agilent Technologies, Inc.

Jan 25, 2024 12:30pm ‐ Jan 25, 2024 1:30pm

Session Speaker:

Untangling the Complexity Within Oligonucleotide Therapeutics  

Jace Jones, University of Maryland School of Pharmacy 

Plenary Session 6: Clearing the Fog: Particle Visibility and Regulatory Updates for Visual Inspection of Parenteral Drug Products and WCBP 2024 Closing Remarks

Jan 25, 2024 3:45pm ‐ Jan 25, 2024 5:30pm

Visual inspection of parenteral drug products is a critical aspect of pharmaceutical development and manufacturing, subject to evolving regulatory standards. This session brings together experts to address the complex landscape of particle visibility and regulatory compliance, with a focus on enhancing product quality and patient safety. The session will provide attendees with a holistic view of the challenges, innovations, and best practices in visual inspection, offering an overview of the regulatory and compendial environment for visual inspection.

Two of the talks will present new data on defining particle visibility: the results from a cross-industry visible particle threshold study, as well as the results from a stand-alone study exploring the opportunities for standardization based on common features of human visual perception.

The session also examines the unique challenges posed by visual inspection for Cell and Gene Therapy (CGT) products, considering whether there are regulatory gaps in this rapidly advancing field.

Finally, the critical role of regulatory agencies in ensuring the safety and efficacy of injectable pharmaceuticals will be discussed, highlighting the collaborative efforts between industry and regulatory bodies in addressing visible particulates in injectable products.

Session Speakers:

The Visual Inspection Regulatory and Compendial Environment: Current Issues and Opportunities
John Shabushnig, Insight Pharma Consulting

Regulatory Considerations for the Assessment of Visible Particulates in Injectable Pharmaceuticals
Rukman De Silva, CDER, FDA

From Blur to Clarity: Definition of Participle Visibility Threshold in Parenteral Drug Products

Felix Nikels, Boehringer Ingelheim Pharma GmbH & Co. KG

Atanas Koulov, Clear Solutions Laboratories

Visual Inspection and Particle Life Cycle Management for CGT Products - Same, Same, but Different... Are There Gaps in the Current Regulations?
Antonio Burazer, Takeda Austria GmbH

Additional Panelists:

Patricia Cash, Global Biotech Experts, LLC

Maryam Mazaheri, I-MAB Biopharma Co., Ltd.