Journal Archive

Foreword


Editorial

Changing the ‘Art’ of Growing Cells into an Industrialized Platform for Commercial-Scale Manufacturing

Spotlight Article

EDITORIAL

Lior Raviv

Latest advances in BIOPROCESSING: overcoming bottlenecks

For the last few years, the cell therapy industry has evolved from a ‘research’ state of mind to a commercialization focus. The recent approvals for Kite, Novartis and Tigenix have boosted the evolutionary process and changed the focus from ‘how to bring the product to approval?’ to ‘what will happen afterwards?’. Based on the proof that cell therapies do work and hold the potential to change the way medicine is practiced, the cell therapy industry is now mature enough to start facing the gaps in translating the manufacturing lines into industrialized platforms.

Published: 6 Nov 2018
DOI: 10.18609/cgti.2018.082
Citation: Cell Gene Therapy Insights; 2018; 4(8), 805-814
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Expert Insight

A question of consistency: biopharma know-how meets bioreactor innovation in upstream bioprocessing

Spotlight Article

Interview

Dr Philipp Nold

LATEST ADVANCES IN BIOPROCESSING: OVERCOMING BOTTLENECKS

Dr. Philipp Nold studied Biology at the Ruhr-University Bochum, Germany and holds a PhD in the same field from the University of Marburg. His diploma thesis about the development of a hematopoietic stem cell niche in a microbioreactor laid the foundation of his knowledge in stem cell biology and cultivation. During his PhD studies at University Hospital of Giessen and Marburg, he successfully established the cultivation of mesenchymal stem cells in a bioreactor in a standardized GMP-conform manner. Based on his work, a manufacturing permit for the utilization of these cells as a therapeutic agent was applied for successfully. Furthermore, he published several scientific articles about stem cells, their capabilities, and their cultivation like “Good manufacturing practice-compliant animal-free expansion of human bone marrow derived mesenchymal stroma cells in a closed hollow-fiber-based bioreactor” published in 2013. In December 2017 Dr. Nold joined the Eppendorf Bioprocess Center as an Infield Application Specialist for stem cells. As part of this, he supports colleagues and customers worldwide with his expertise in stem cell bioprocessing and applications. This includes, among others, trainings, presentations at conferences, and webinars.

DOI:10.18609/cgti.2018.058
Citation: Cell Gene Therapy Insights 2018; 4(8),601-606.
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Overcoming Bottlenecks in AAV Manufacturing for Gene Therapy

Spotlight Article

EXPERT INSIGHT

Jacob Smith, Josh Grieger & R Jude Samulski

Latest advances in BIOPROCESSING: overcoming bottlenecks

Recombinant adeno-associated viral vectors (rAAV) have emerged as one of the most powerful tools for gene delivery to treat human disease. Significant advancements in vector development and manufacturing methods, along with promising results in the clinic, has initiated an extraordinary interest in drug development for gene therapies and commercialization. It is an exciting time for AAV-mediated gene therapy; in 2012, the field witnessed the ap-proved use of the gene therapy product, Glybera, for the treatment of lipoprotein lipase deficiency by the European Medicines Agency, and in 2017, the approval of Luxturna, for the treatment of Leber’s Congenital Amaurosis by the US FDA, thus arriving at the threshold of gene therapies reaching patients. Still, with an increasing number of gene therapy programs and a growing de-mand for both pre-clinical and clinical grade vector, the industry must be prepared to implement practical solutions to address the manufacturing bottlenecks and rigorous product characterization needed to meet higher regulatory requirements earlier in clinical development in order to efficiently advance and market these products. Below is an overview of the current status of rAAV vec-tor production from academic to contract manufacturing organization (CMO).

Submitted for peer review: 23 Aug 2018 Published: 6 Nov 2018
DOI: 10.18609/cgti.2018.083
Citation: Cell Gene Therapy Insights2018; 4(8), 815-827.
Open access

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The Critical Role of Testing to Ensure Product Quality

Spotlight Article

Interview

Kevin Barfield, Sara Dionne & Ronnie Aga

Latest Advances in Bioprocessing: Overcoming Bottlenecks

KEVIN BARFIELD serves as the President of VRL-Eurofins. Mr. Barfield has more than 20 years of progressively responsible experience leading companies through start-up, turnaround and growth modes. His understanding of the laboratory industry encompasses operations, business development, marketing, customer service, logistics, direct sales, and quality and regulatory compliance. Among his significant previous positions, Kevin served as President and Chief Operating Officer of Laboratories at Bonfils, a Denver based reference laboratory performing donor eligibility testing for the transplant community. Kevin holds a Bachelor of Science degree in Health Sciences and Bachelor of Business Administration degree in Marketing. He is also a registered Medical Technologist, receiving his training from the United States Air Force and Midwestern University.

SARA DIONNE serves as VRL-Eurofins Senior Laboratory Director. She received her Ph.D. in the field of Microbiology & Immunology and she started her career as Assistant Professor at the University of Arizona, Department of Medicine. Dr. Dionne is credentialed through the American Board of Histocompatibility and Immunogenetics and is the Director of the Immunogenentics Department, a position she has held since 2011. Dr. Dionne has worked in the field of laboratory testing for HCT/Ps for over a decade specializing in Infectious Disease and Microbiology screening of donors and biological products. Dr. Dionne provides customized consultations for VRL-Eurofins clinical partners and is responsible for operational oversight of all VRL laboratories.

RONNIE AGA serves as the Executive Director for VRL-Eurofins. Mr. Aga graduated in 1990 from University of North Texas with a Bachelors of Science degree in Biology. Since graduation, he has spent his entire career in transplant laboratory medicine. During the last 27 years, Ronnie performed multiple testing assays, lead the client services and specimen management departments, directed sales activities and managed the client supply and logistics support services.

DOI: 10.18609/cgti.2018.058
Citation: Cell Gene Therapy Insights 2018; 4(8), 827-833.
Open access

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Tissue Manufacturing by Bioprinting: Challenges & Opportunities

Spotlight Article

EXPERT INSIGHT

Fabien Guillemot, Laurence Hutter, Bruno Brisson, Delphine Fayol & Bertrand Viellerobe

Latest advances in BIOPROCESSING: overcoming bottlenecks

Despite substantial investments to meet clinical and commercial expectations, and while scientific achievements at the preclinical research stage have sometimes been impressive, scaffold-based tissue engineering approaches are struggling to find the way to therapeutic and industrial success. The main challenges for the manufacture of tissue-engineered Advance Therapy Medicinal Products (ATMPs) concern the improvement of the standardization of manufacturing processes, tissue functionality and cost–effectiveness and profitability of related treatments. Based on our experience in the field of bioprinting, we discuss how this technology – thanks to its characteristics resulting from the convergence of automation, biology and digital technology – should make it possible to overcome current tissue manufacturing bottlenecks and also provide new opportunities.

Submitted for review: Aug 18 2018 Published: Nov 6 2018
DOI: 10.18609/cgti.2018.081
Citation: Cell Gene Therapy Insights; 2018; 4(8), 781-790
Open access

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Next-Generation Stem Cell Expansion Technologies

Spotlight Article

EXPERT INSIGHT

Carlos AV Rodrigues, Diogo ES Nogueira & Joaquim MS Cabral

Latest Advances in Bioprocessing: Overcoming Bottlenecks

Cell therapies, where living cells are used as therapeutic agents, either to regenerate tissues affected by disease or to act as delivery agents for secreted factors, are commonly based on the action of stem cells or their differentiated progeny. These cells also have great potential for drug discovery, providing access to inaccessible patient-specific cell types. However, stem cells, which are the most important ‘raw material’ for these applications, have to be expanded in vitro to generate the high quantities of cells that are expected to be required. Decades of research and application of bioreactor engineering concepts have provided a solid foundation for use of advanced culture technologies for stem cell manufacturing. The particular challenges of producing high quantities of functional cells are discussed here as well as innovative approaches and technologies that may revolutionize the field.

Submitted for peer review: 31st Jul 2018
Published: 31st Oct 2018
DOI: DOI: 10.18609/cgti.2018.076

Citation: Cell Gene Therapy Insights 4(8), 791-804
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Streamlining data management & process analytics for the manufacturing of cell & gene therapies

Spotlight Article

EXPERT INSIGHT

Sébastien de Bournonville, Toon Lambrechts, Thomas Pinna, Ioannis Papantoniou & Jean-Marie Aerts

LATEST ADVANCES IN BIOPROCESSING:OVERCOMING BOTTLENECKS

With a likely increase in production volumes of cell and gene therapies, and the more widespread implementation of Quality by Design principles, the amount of process data to manage (i.e. collect, validate, analyze and interpret) will increase significantly. Data guides the manufacturing operations, it allows monitoring and control of the process, and it assures product quality and regulatory compliance. More recently big data, artificial intelligence and machine learning techniques promise a surge in data-driven insights, affecting nearly all aspects of R&D and manufacturing of cell and gene therapies. However, without proper tools and solid data foundations, data management will constitute a bottleneck in the production of these novel therapies. The aim of this article is to discuss steps needed in order to address the data management challenge.

Submitted for peer review: 16 Aug 2018 Published: 6 Nov 2018
DOI: 10.18609/cgti.2018.068

Citation: Cell Gene Therapy Insights 4(8), 695-704
Open access

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Innovator Insight


Development of purification steps for several AAV serotypes using POROS™ CaptureSelect™ AAVX affinity chromatography

Spotlight Article

Innovator Insight

M Toueille, L Dejoint, E Attebi, J Cartigny, C Rasle, S Potier, S Rundwasser, L Guianvarc’h, C Lebec & M Hebben

latest advances in BIOPROCESSING: overcoming bottlenecks

Genethon is an integrated R&D centre created in 1990 by the French Association of Muscular Dystrophy. The Centre performs both gene therapy translational research and clinical development, with a specific focus on rare genetic diseases such as neuromuscular disorders, immuno-deficiencies, eye diseases and liver diseases. The two main vector platforms used at Genethon are AAV and lentiviral vectors.

Published: Sept 18 2018
DOI: 10.18609/cgti.2018.061
Citation: Cell Gene Therapy Insights 4(7), 637-645
Open access

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