Bringing you the latest cutting-edge research and commentary in bioscience.

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 5 Issue 10

Commercial Insights
Interview

Mitigating raw materials risk during preclinical-clinical transitioning

Interview

Adam Hostetler & Gerry Maybach


Supply Chain Focus: Biologistics Strategy for Scale-Up


Adam Hostetler earned his BA in business management and economics and BSc in biology from the University of Pittsburgh at Johnstown and his MBA from the University of Pittsburgh. He currently serves as Director of Operations for Cook MyoSite Incorporated, a Cook Group company. Cook MyoSite is currently conducting several clinical studies for their autologous cell-therapy treatment. Adam is responsible for providing strategic direction to the Manufacturing Operations, Technical Services, Customer Logistics and Finance units, as well as the Project Management and Operational Excellence Office. Adam has been with Cook MyoSite for over 11 years, where much of his focus has been directed toward establishing a scalable supply chain for commercialization of Cook MyoSite’s cell therapy treatments.

Gerry Maybach earned his BA in Chemistry from Oswego State University in Oswego, New York. He currently serves as the Plant Manager for the two facilities that comprise Cook MyoSite Incorporated, a Cook Group company. Gerry is responsible for translating the strategic direction for the Manufacturing Operations into tactical and executable plans across the Supply Chain, Planning and Production, Warehouse Operations, Facility Engineering and EHS, and Metrology departments. Gerry has been with Cook MyoSite for almost 2 years focusing on the execution of lean manufacturing strategies while driving continuous improvement in business and supply chain continuity across the operations. With over 20 years of global experience in the biopharmaceutical industry, Gerry has held senior leadership roles in engineering and operations management, as well as leading contract development and manufacturing operations (CDMO).

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Building flexibility into GMP CAR T cell therapy manufacture

Spotlight Article

Interview

Christin Tischner

Decentralized Manufacture

Christin Tischner currently works at Miltenyi Biotec as a Clinical Supply Chain Manager. This interview was conducted when Christin was working in her previous role as Tech Transfer Project Manager for CAR-T Cell Manufacturing at Cellex. She is a Molecular Biologist by training and obtained a PhD in Genetics from the University of Cologne.

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Identifying and mitigating risks in the viral vector supply chain

Interview

Claire Wartel & Ulises Villavicencio

Decentralized Manufacture


VECTOR CHANNEL: RAW MATERIALS


Claire Wartel has a PhD in Molecular Pharmacology and Pharmacochemistry from the University of Strasbourg (France, 1999). After several post-docs in different prestigious universities, Claire joined Polyplustransfection in 2004 as Cell Biology project leader and participated in the development of our flagship product jetPRIME®. Claire has taken Quality responsibility since 2007, and nowadays manages the department of Quality and Regulatory Affairs.

Ulises Villavicencio attended California State University, Long Beach (CSULB) where he earned a Bachelor’s Degree in Anthropology and focused on Archaeometry. Ulises participated in numerous archaeological research projects, domestic and international, and assisted in various analytical projects at CSULB’s Institute for Integrative Research in Materials, Environments, and Societies to further analytical research in Archaeometry via chemistry-based applications. Ulises applied his education and experience in Quality test environments where he then expanded into the auditing field in 2015. Since then, Ulises has focused his attention on improving Quality and Compliance programs for the security and assurance of patient safety, where he manages the Supplier Qualification program in his current role.

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Major bioprocessing challenges & considerations with gene therapies and ex vivo gene-edited cell therapy

Interview

Andy Ramelmeier

Decentralized Manufacture


VECTOR CHANNEL: RAW MATERIALS


Andy Ramelmeier currently serves as Executive Vice President and Chief Manufacturing and Quality Officer and is responsible for Technical Operations at Sangamo, including manufacturing, quality supply chain, and process and analytical development. Dr Ramelmeier has 25 years of experience in the biopharmaceutical industry, developing and transferring biological processes, designing and building manufacturing facilities, and directing contract manufacturers as well as internal manufacturing operations. Prior to joining Sangamo in January 2018, he served as Senior Vice President, Technical Operations at Portola Pharmaceuticals, Inc., where he was responsible for tech transfer, bulk and drug product manufacturing, technical support and supply chain of Portola’s pipeline products. From 2006 to 2014, Dr Ramelmeier served as Vice President, Manufacturing, Process Sciences and Facilities at BioMarin, overseeing multiple commercial biologics products, clinical pipeline, and facilities in Novato, CA, and Shanbally, Ireland. Earlier in his career, he held roles of increasing responsibility at Johnson & Johnson and Merck. Prior to joining industry, Dr Ramelmeier conducted post-doctoral work in Germany. He received a BSc in Chemical Engineering from Johns Hopkins and his PhD in Chemical Engineering from the University of California, Berkeley.

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Challenges in viral vector raw materials procurement & management

Interview

Kathleen Szczur

Decentralized Manufacture


VECTOR CHANNEL: RAW MATERIALS


Kathleen Szczur, Senior Specialist in the Vector Production Group at Cincinnati Children’s Hospital Medical Center (CCHMC), has over 21 years of experience in both basic science research and Phase 1/2 GMP manufacturing. She began her career in production of GMP clinical supplies and analytical chemistry at a small firm working with biodegradable polymers to develop long-acting delivery systems of various molecules. She continued her career researching immune function at Shriners Burns Hospital, Cincinnati, OH, in the lab of Cora K Ogle, PhD and hematopoiesis with the Experimental Hematology and Cancer Biology group at CCHMC in the lab of David A Williams, MD and Marie-Dominique Filippi, PhD. Her current position involves manufacturing of viral vectors for both academic and commercial customers to advance the field of gene therapy.

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Identifying and mitigating risks in the viral vector supply chain

Interview

Claire Wartel & Ulises Villavicencio

Decentralized Manufacture


VECTOR CHANNEL: RAW MATERIALS


Claire Wartel has a PhD in Molecular Pharmacology and Pharmacochemistry from the University of Strasbourg (France, 1999). After several post-docs in different prestigious universities, Claire joined Polyplustransfection in 2004 as Cell Biology project leader and participated in the development of our flagship product jetPRIME®. Claire has taken Quality responsibility since 2007, and nowadays manages the department of Quality and Regulatory Affairs.

Ulises Villavicencio attended California State University, Long Beach (CSULB) where he earned a Bachelor’s Degree in Anthropology and focused on Archaeometry. Ulises participated in numerous archaeological research projects, domestic and international, and assisted in various analytical projects at CSULB’s Institute for Integrative Research in Materials, Environments, and Societies to further analytical research in Archaeometry via chemistry-based applications. Ulises applied his education and experience in Quality test environments where he then expanded into the auditing field in 2015. Since then, Ulises has focused his attention on improving Quality and Compliance programs for the security and assurance of patient safety, where he manages the Supplier Qualification program in his current role.

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Challenges in demonstrating comparability in the decentralized manufacturing environment

Spotlight Article

Interview

Jonathan Campbell

Decentralized Manufacture

Jonathan Campbell obtained his PhD from the IRC in Biomedical Materials at Queen Mary University of London investigating the role of mechanics on MSC osteochondral differentiation before taking up post-doctoral positions at both QMUL and Cambridge, optimising biomaterial scaffolds for tissue development and disease modelling applications. In his current role at the National Measurement Laboratory hosted at LGC Ltd, he has responsibility for delivering cell measurement research and standards projects and inputting more widely to the strategic objectives of the NML. He is active on several UK and international standardisation committees including ISO TC276.

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Pros and cons of point of care manufacture of cellular cancer immunotherapies

Spotlight Article

Interview

Linda Kelley

Decentralized Manufacture

Dr Linda Kelley, Cell Therapy Facility Director, is a Senior Member at Moffitt Cancer Center and Professor at the University of South Florida. Dr Kelley has provided leadership for cellular therapy facilities for over 20 years at three institutions, University of Utah, Dana Farber Cancer Institute and Moffitt Cancer Center. She received graduate and post-doctoral training in immunology and hematology from Vanderbilt University, Nashville, TN. Her scientific career evolved from a fundamental interest in immunological mechanisms of T-lymphocyte function, growth mechanisms of hematopoietic stem and progenitor cells and molecular changes associated with malignant transformation. Knowledge of the hematopoietic system led to an interest in stem cell biology and therapies. As director of the Cell Therapy Facility at the University of Utah from 1994 to 2011, she was responsible for developing and expanding a Cell Therapy and Regenerative Medicine Laboratory. During her tenure she was responsible for pre-clinical and clinical cell therapy product development to support IND applications for the production of allogeneic mesenchymal stromal cells (MSC), autologous bone marrow-derived mononuclear cells and allogeneic fetal-derived oligodendrocytes. As director of the Cell Manipulation Core Facility at the Dana Farber Cancer Institute at Harvard from 2011 to 2012, she oversaw management of 20 FDA-approved INDs for the manufacture of gene-modified CD34+ cells, tumor cell vaccines, dendritic cells, MSCs and others. As director of the Cell Therapy Facility at Moffitt Cancer Center, she oversees 22 active INDs for a variety of cell therapy products largely to support immunotherapy for adult and pediatric patients. She currently serves as the Principal Investigator for Production Assistance for Cellular Therapies (PACT) – Cell Processing Facilities to perform pre-clinical cell therapy product development in collaboration with NHLBI and other PACT Centers and as Core Laboratory Technical Director for the Moffitt Cancer Center Support Grant. Dr Kelley excels at bridging the gap between laboratory-based discoveries and new therapies for patients.

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Perspective

ATMP raw materials: ‘the plasmid conundrum’

Perspective

Alan Griffith

Decentralized Manufacture


VECTOR CHANNEL: RAW MATERIALS


Plasmids and plasmid DNA (pDNA) have been key components in recombinant DNA molecular biology for decades. One of their uses as a precursor raw material represents a cornerstone of viral vector ATMP manufacturing. Here we provide a synopsis with regard to their own manufacturing lifecycle, limitations, demand, regulatory expectations and supply chain, as more and more companies join the gene and cell therapy clinical trial races to market in this fast-paced sector.

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Expert Insight

Decentralized manufacture of cell therapies: the challenge of operational modeling

Spotlight Article

Expert Insight

Nicholas Medcalf

Decentralized Manufacture

Cell therapies may often benefit from a decentralized manufacturing approach. In order to choose from amongst the possible options and to coordinate the activity of the various agents in the chain of value the industry needs reliable decision-making tools. Three tools are offered here that will help with evaluation of this route. Tools for enterprise modeling, inclusive cost analysis and timing of research investment are introduced, showing the potential for applying systems engineering to decentralized manufacturing. The case is made for initiatives to encourage communities of practice and data-sharing for operational models and unit operations.

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Cell manufacturability

Spotlight Article

Expert Insight

Masahiro Kino-oka, Manabu Mizutani & Nicholas Medcalf

Decentralized Manufacture

The manufacturers of cell therapy and regenerative medicine products must design manufacturing operations to deliver the required level of stability. Process consistency, maintenance of the aseptic environment (to prevent contamination), assurance of line separation (to prevent cross-contamination and operational confusion), containment (for avoidance of cross-contamination), and their routine management must be taken into account. In this article, we propose the concept of ‘cell manufacturability’ for process development in order to assist the design of cell manufacturing processes.

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Implementation of advanced therapy medicinal products: a UK pharmacist’s perspective

Spotlight Article

Expert Insight

Anne Black

Decentralized Manufacture

Advanced therapy medicinal products (ATMP) have introduced a new area of specialism to the clinical pharmacy workforce. The author will explore the role of the pharmacist in the UK in the delivery of cell-based medicines, emphasizing governance and clinical requirements, and recognizing that operationalizing ATMPs requires a collaborative multidisciplinary approach to ensure that the medicines are optimized for patients. This involves ensuring that whilst appropriate pharmacovigilance and pharmaceutical procedures are in place, handling is undertaken by a workforce that is trained and competent in the handling of cellular products.

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Regulatory considerations for decentralized manufacture of ATMPs

Spotlight Article

Expert Insight

Alison Wilson & Alexis Cockroft

Decentralized Manufacture

Decentralized manufacture (DCM) has the potential to facilitate uptake of advanced therapy medicinal products (ATMPs) within the EU. The new GMP guideline for ATMPs contains welcome new flexibility in regard to DCM and also in relation to use of automated equipment. However regulatory challenges extend beyond GMP issues, including questions of comparability of product manufactured at multiple hospital sites and mechanisms for introduction of new sites within the current variations framework. The need for additional guidance from regulators is discussed.

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Decentralized manufacturing and institutional readiness: adoption as a distributed process

Spotlight Article

Expert Insight

Andrew Webster

Decentralized Manufacture

The model of decentralized manufacture is said to offer a new approach for more effective translation of cell and gene therapies to the clinic (and indeed in other sectors). Much of this depends on ensuring that key processes such as scalability and traceability are well-understood and properly managed as products move through to the clinic. The adoption of these therapies will require the creation of a novel trans-organizational innovation space. The latter can be better understood through deploying the social science model of institutional readiness to focus attention on the specific capacities that are needed to create not merely working but workable therapies, those that make sense in the clinical environment. This article outlines the model of institutional readiness, comparing it with the linear and primarily technically-based model of ‘technology readiness levels’, showing how it can help anticipate the specific capacities needed to build a new (decentralized) innovation space.

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Research Article

Regenerative medicine as a disruptive technology: implications for manufacturing & clinical adoption

Spotlight Article

Research Article

Geoffrey Banda, Joyce Tait & James Mittra

Decentralized Manufacture

Although regenerative medicine has been described as a disruptive innovation, there has been little critical enquiry into the nature and location of the disruption. This paper, based on ten cases in the UK, analyses the nature of disruption for allogeneic and autologous therapies in terms of manufacturing, distribution and adoption in clinical settings. We discuss the challenges of dealing with inherent variability in living systems and how this necessitates co-evolution of technologies and innovations. We propose that understanding of the distinction between disruptive and incremental innovation, and of the nature, extent and location of the disruption across sectoral value chains, can help to guide company innovation strategies and government innovation support policies for regenerative medicine, as already proposed for industrial biotechnology.

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