ARM has spent nearly four years on a mission to answer the question “What is the biggest challenge to cell and gene CMC and how can ARM address it?”. Many of the hurdles to streamlined, cost effective manufacture of cell and gene therapy products derive from a lack of standardized methodologies and training around CMC programs. Borrowing from the ‘A-Mab’ model created for the monoclonal antibody sector, ARM members have produced a similar document titled A-Gene, a case study-based guide to integrating QbD principles in gene therapy CMC programs.
In conjunction with the A-Gene document, ARM hosted a six-part live webinar series highlighting specific A-Gene chapters which culminated in a half-day virtual workshop in June of 2022.
A-Gene Virtual Workshop
ARM hosted a half-day virtual workshop focusing on the best practices for the gene therapy sector put forth in the A-Gene document. This workshop covered a variety of topics including downstream processing, comparability and the development of functional potency assays.
Mike Lehmicke, Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine (ARM)
Downstream Processing Using QbD
Michael Mercaldi, Ph.D., Vice President and Head of CMC, Purification and Drug Product Sciences, Oxford Biomedica Solutions
Comparability During the Various Stages of Development
Mo Heidaran, Ph.D., Head of Translational and Regulatory Strategy, GC Therapeutics
Tim Kelly, CEO, Oxford Biomedica Solutions
Paul McCormac, Ph.D., Chief Technical Officer, LEXEO Therapeutics
Kevin Whittlesey, Ph.D., Principal, Dark Horse Consulting Group
Development of Functional Potency Assays
Adam Davis, Ph.D., Senior Director, Analytical Development, Forge Biologics
Rebecca Raig, Scientist I, Product Development, Forge Biologics
Adopting a QbD Mindset for Designing, Building and Validating a Commercial Gene Therapy Facility
Samir Acharya, Associate Director of Process Development, Andelyn Biosciences
Cyrill Kellerhals, Head of Manufacturing, Andelyn Biosciences
Kenneth LaRiviere, Head of Engineering, Andelyn Biosciences
Digital Strategies for Successful Tech Transfer
Ken Forman, Senior Director of Product Strategy, IDBS
Process Development Using Quality by Design (QbD) Principles
Jessie Sun, Ph.D., Director, Upstream Process Development, Pharmaceutical Development, Ultragenyx Gene Therapy
James Warren, Ph.D., Vice President, Pharmaceutical Development, Ultragenyx Gene Therapy
This webinar discusses process development and how it aims to establish and characterize a manufacturing process that can be scaled up to manufacture-size batches while continuing to yield a consistent, quality product. Various risk-based approaches are utilized to ensure that the process stays within appropriate limits and meets all safety and quality benchmarks.
Process Control Strategy
Althea Micklewright, Director, PSQA Business Excellence Lead, Pfizer
Iryna Sanders, Ph.D., Senior Principal Scientist, Pfizer
This webinar discusses how a comprehensive control strategy for pharmaceutical products – including gene therapies – is the key to achieving process consistency, product quality, safety, and efficacy. The speakers describe the approaches to developing a comprehensive control strategy and provide an overview of the key elements of a control strategy for a gene therapy product.
Download this webinar’s Q&A transcript here.
Shamik Sharma, Ph.D., Director, Molecular Biology and Upstream Process Development, Voyager Therapeutics
This webinar discusses the process overview of the upstream production of an AAV vector using an HEK 293 cell line. The speaker describes the Quality by Design (QbD) approach to process understanding and scaling.
A recording of this webinar will not be made available.
Jeffrey Hung, Ph.D., General Manager, Vigene Biosciences, a Charles River Company
This webinar discusses the vector manufacturing process and how it culminates with the formulation and vialing of the purified vector product. While this stage may come temporally at the end of the process, it is of the utmost importance to begin the gene therapy development process while considering the “end goal”. This webinar outlines the best practices for fill-finish, formulation, characterization, CCIT, and long-term storage and stability that can be used to develop a robust final gene therapy product.
Mo Heidaran, Ph.D., Vice President, Technical, Parexel International
This webinar provides guidance to sponsors and developers on the best practices and recommendations in managing manufacturing changes during early development by establishing basic concepts in comparability appropriate for gene therapy products, with a focus on adeno-associated virus (AAV)–based products.
Alexandra Beumer Sassi, Ph.D., Pharm.D., Senior Director, Chemistry, Manufacturing and Controls, Voisin Consulting Life Sciences
Saroj Ramdas, Vice President, CMC Regulatory and Technical Strategy, Amicus Therapeutics
This webinar will discuss the regulatory frameworks in the United States, European Union, and Japan. It will also provide guidance to sponsors on the best practices and recommendations for interactions with the Health Authorities illustrating with some case studies for gene therapy products.