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

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

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Journal

Welcome to the latest issue of Cell and Gene Therapy Insights – the open access, peer-reviewed publication that brings you the latest insight and commentary from leading experts from academia and industry.

Foreword

Managing starting materials collection and logistics at the clinical point of care

Foreword

John DM Campbell


Supply Chain Focus: Materials Collection & Logistics at the Clinical Point of Care


John DM Campbell is Associate Director of Tissues, Cells and Advanced Therapeutics at the Scottish National Blood Transfusion Service (SNBTS) in Edinburgh. He completed his PhD in Pathology at Edinburgh in 1995, and has worked in the cellular therapy field for 30 years in the academic, industrial and healthcare sectors.

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Expert Roundtable

Expert Roundtable: Scale-up considerations for improved yield in upstream viral vector production

Spotlight Article

Expert Roundtable

Sven Ansorge, Mike Burnham, Michael Kelly, Ruth McDermott, Peter Jones

2020 Vision



Sven Ansorge
Director of Manufacturing at ExCellThera Inc.

Sven holds a PhD in Chemical Engineering and has led teams and managed projects within academic and industrial/GMP manufacturing environments. He is specialized in bioprocessing for the production of viral vectors, vaccines and recombinant proteins.

Mike Burnham
Director of the Vector Development department at Tmunity Therapeutics Inc.

Mike has over 25 years’ of biosafety and bioprocessing expertise, and is currently working to establish Tmunity’s lentiviral vector manufacturing platform. Prior to joining the team, Mike worked at WuXi AppTec, serving in multiple roles in their Process Development, Viral Production/Purification and Viral Clearance departments.

Michael Kelly
Vice President of Process Development at Avrobio

Avrobio is a clinical stage company developing gene therapies for rare diseases. Michael has over 20 years of experience in the field of gene therapy. During this time, Mike has been responsible for various aspects of research, development and clinical manufacturing for a number of lentiviral, adenoviral and AAV-based therapeutics.

Ruth McDermott
Segment Marketing Manager at Sartorius

Ruth has 20 years of business and market development experience within the life sciences industry, including roles in technology and IP commercialization. Ruth’s technical knowledge has been built on a background of R&D in cell bioprocessing, stem cells and gene therapy. Ruth holds a PhD in Cell Biology and an MBA.

Peter Jones
Head of Operational Strategy at Oxford Biomedica

Peter has over 30 years’ experience in the biopharmaceutical industry. He joined Oxford Biomedica in 2011, providing technical and scientific leadership. In his current role, he is responsible for overseeing strategic and operational planning to develop the Company’s capabilities and capacity to support future business growth. Peter is a Fellow of the Institution of Chemical Engineers and the Royal Academy of Engineering Visiting Professor in Manufacturing of Advanced Therapies at Aston University.

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Scale-up considerations for improved yield in upstream viral vector production

Spotlight Article

Expert Roundtable

Sven Ansorge, Mike Burnham, Michael Kelly, Ruth McDermott, Peter Jones

2020 Vision


Sven Ansorge
Director of Manufacturing at ExCellThera Inc.
Sven holds a PhD in Chemical Engineering and has led teams and managed projects within academic and industrial/GMP manufacturing environments. He is specialized in bioprocessing for the production of viral vectors, vaccines and recombinant proteins.


Mike Burnham
Director of the Vector Development department at Tmunity Therapeutics Inc.
Mike has over 25 years’ of biosafety and bioprocessing expertise, and is currently working to establish Tmunity’s lentiviral vector manufacturing platform. Prior to joining the team, Mike worked at WuXi AppTec, serving in multiple roles in their Process Development, Viral Production/Purification and Viral Clearance departments.


Michael Kelly
Vice President of Process Development at Avrobio
Avrobio is a clinical stage company developing gene therapies for rare diseases. Michael has over 20 years of experience in the field of gene therapy. During this time, Mike has been responsible for various aspects of research, development and clinical manufacturing for a number of lentiviral, adenoviral and AAV-based therapeutics.

Ruth McDermott
Segment Marketing Manager at Sartorius
Ruth has 20 years of business and market development experience within the life sciences industry, including roles in technology and IP commercialization. Ruth’s technical knowledge has been built on a background of R&D in cell bioprocessing, stem cells and gene therapy. Ruth holds a PhD in Cell Biology and an MBA.


Peter Jones
Head of Operational Strategy at Oxford Biomedica
Peter has over 30 years’ experience in the biopharmaceutical industry. He joined Oxford Biomedica in 2011, providing technical and scientific leadership. In his current role, he is responsible for overseeing strategic and operational planning to develop the Company’s capabilities and capacity to support future business growth. Peter is a Fellow of the Institution of Chemical Engineers and the Royal Academy of Engineering Visiting Professor in Manufacturing of Advanced Therapies at Aston University.

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Commercial Insights
Interview

Introducing flexibility to automation to unleash the power of biology – Podcast

Spotlight Article

Interview

Markus Gershater

2020 Vision

Markus co-founded Synthace after working as a Research Associate in Synthetic Biology at University College London where he developed novel biosynthesis methods using pathway engineering. Prior to UCL, he was a Biotransformation Scientist at Novacta Biosystems working as part of the industrial biotechnology group that conducted more than 90 contract research projects for over 20 clients. Markus has a PhD in Plant Biochemistry from Durham.

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Introducing flexibility to automation to unleash the power of biology

Spotlight Article

Interview

Markus Gershater

2020 Vision

Markus co-founded Synthace after working as a Research Associate in Synthetic Biology at University College London where he developed novel biosynthesis methods using pathway engineering. Prior to UCL, he was a Biotransformation Scientist at Novacta Biosystems working as part of the industrial biotechnology group that conducted more than 90 contract research projects for over 20 clients. Markus has a PhD in Plant Biochemistry from Durham.

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Analyzing key ATMP talking points through IP and regulatory lenses

Spotlight Article

Interview

Beatriz San Martin & Jackie Mulryne

2020 Vision

Beatriz San Martin is an intellectual property specialist focusing her practice on the life sciences sector and emerging/disruptive technologies. She has significant experience handling cases before the UK Courts and the Court of Justice of the European Union, including high-profile European litigation. Beatriz was formerly a scientist with a Wellcome Trust PhD and a postdoctoral from the University of Cambridge specializing in genetics, cellular, and molecular biology. She is a member of the UK BioIndustry Association Cell and Gene Therapy Advisory Committee and Engineering Biology Advisory Committee.

Jackie Mulryne is a Partner at Arnold & Porter, focusing on regulatory and contentious matters for life sciences companies. She has advised a range of clients on the development and launch of innovative products, and assisted with navigating the regulatory landscape for novel cell and gene therapy products.

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Advancing iPSC-derived NK cell therapy

Spotlight Article

Interview

Bahram Valamehr

2020 Vision

Bahram (Bob) Valamehr is the Chief Development Officer at Fate Therapeutics, overseeing the company’s early development activities including ‘off-the-shelf’ cell therapy product candidates derived from the company’s induced pluripotent stem cell platform. Previously, Dr Valamehr was the Vice President of Cancer Immunotherapy at Fate and prior to that played ey scientific roles at Amgen, the Center for Cell Control (a NIH Nanomedicine Development Center) and the Broad Stem Cell Research Center developing novel methods to control pluripotency, to modulate stem cell fate including hematopoiesis and to better understand cellular signaling pathways associated with cancer. He has co-authored numerous studies and patents related to stem cell biology, oncology and materials science. Dr Valamehr received his PhD from the Department of Molecular and Medical Pharmacology at UCLA, his MBA from Pepperdine University and his BS from the Department of Chemistry and Biochemistry at UCLA.

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Oncology and beyond: looking to the future of CAR-T and autologous cell therapy

Spotlight Article

Interview

Stephan Grupp

2020 Vision

Stephan Grupp is the Chief of the Cellular Therapy and Transplant Section, Director of the Cancer Immunotherapy Program, and Medical Director of The Cell and Gene Therapy Lab at the Children’s Hospital of Philadelphia (CHOP), as well as the Yetta Dietch Novotny Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine. Dr Grupp graduated from the University of Cincinnati after completing the MD/PhD program with a PhD in Immunology. He completed pediatric residency at the Boston Children’s Hospital, followed by a fellowship in Pediatric Hematology/Oncology at the Dana Farber Cancer Institute and postdoctoral work in Immunology at Harvard University. He then joined the faculty at Harvard University until 1996, when he came to CHOP. His primary area of clinical research is the use of CAR T and other engineered cell therapies in relapsed pediatric cancers. He led the pediatric ALL trials of CTL019 (now approved as Kymriah), including the largest and most successful engineered T-cell therapy clinical trial conducted to date, as well as the global registration trial for CTL019. As a result of this work, he presented the Clinical Perspective at the July 2018 FDA ODAC meeting, at which reviewers voted 10–0 for recommendation of approval for Kymriah in pediatric ALL. His primary laboratory interest is the development of new cell therapy treatments for pediatric cancers. Dr Grupp was elected to the National Academy of Medicine in 2019.

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Bioprocess decisional tools to enable cell and gene therapy commercialization

Spotlight Article

Interview

Suzanne Farid

2020 Vision

Suzanne Farid is Professor of Bioprocess Systems Engineering at the Advanced Centre for Biochemical Engineering at University College London (UCL) and Deputy Head of Department (Education). She is Co-Director of the Future Targeted Healthcare Manufacturing Hub in collaboration with industrial and academic consortia to revolutionize the delivery of cost-effective stratified protein-based and personalized cell-based therapies to patients. She is also Director of the UCL-AstraZeneca Centre of Excellence. She leads research on ‘Decisional Tools’ to facilitate cost-effective bioprocess design, capacity planning, R&D portfolio management, root cause analysis and manufacturability assessments for biopharmaceuticals ranging from mAbs to cell and gene therapies. She sits on the ISCT Business Models and Investment Sub Committee, UK BioIndustry Association Manufacturing Advisory Committee and is a Fellow of the IChemE. She obtained her Bachelor’s and PhD degrees in Biochemical Engineering from UCL.

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Evolving the supply chain to counter key challenges in apheresis and leukapheresis collection

Interview

Peter Olagunju

2020 Vision


Supply Chain Focus: Materials Collection & Logistics at the Clinical Point of Care


Peter Olagunju is the Vice President of Patient Operations with bluebird bio. In this role, Peter has responsibility for managing the CMC related Centers of Excellence (COE) interfaces including the collection of patient cells, manufacture of drug product, and operational execution of patient treatments. Prior to bluebird bio, Peter most recently served as Senior Director, Global Technical Operations at Valeant (through the acquisition of Dendreon). He was at Dendreon for roughly 5 years where he managed the US and EU manufacturing operations and supply chain, including contract manufacturers, external testing sites, and the apheresis network for those regions. Prior to Dendreon, Peter had leadership positions within the quality function at a monoclonal antibody organization (ZymoGenetics) that was acquired by BMS in 2011. Peter did his undergraduate studies at the University of Illinois and completed an MBA program at the Foster school of Business (University of Washington).

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Brave new world – a new decade in allogeneic cellular immunotherapy

Spotlight Article

Interview

David Sourdive

2020 Vision

David Sourdive is a co-founder of Cellectis and joined the Board of Directors in 2000. Dr. Sourdive combines a strong scientific expertise with experience in managing industrial programs bringing innovative technologies to industrial fruition. He served as Executive Vice President, Corporate Development, from 2008 to 2016 and as Executive Vice President, Technical Operations until July, 2019. In addition to his role at Cellectis, Dr. Sourdive has also served on the board of directors of the Mediterranean Institute for Life Sciences. David Sourdive graduated from École Polytechnique, received his Ph.D. in molecular virology at Institut Pasteur and completed a research fellowship in the Emory University Department of Microbiology and Immunology. His management training is from the HEC (Challenge +) and his decade-long experience in industrial program management was acquired at the French Department of Defense (DGA) prior to Cellectis’ inception.

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Where will synthetic biology take cell & gene therapy?

Spotlight Article

Interview

Tim Lu

2020 Vision

Tim Lu is a pioneer in synthetic biology and an Associate Professor at MIT. He received his MD/PhD from MIT and Harvard. He is a co-founder of Synlogic (NASDAQ: SYBX), Tango Therapeutics, Sample6, Eligo, BiomX, and Engine Biosciences. He has received the NIH New Innovator Award, the US President Early Career Award for Scientists and Engineers, MIT Technology Review’s TR35, Navy and Army Young Investigator Prizes, and others.

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Next steps for the iPSC and tissue engineering fields

Spotlight Article

Interview

Ioannis Papantoniou

2020 Vision

Ioannis Papantoniou is a Chemical Engineer by training (graduated from the University of Patras, Greece) and is currently a Principal Investigator at the Institute of Chemical Engineering Sciences at the Foundation of Research&Technology-Hellas and a visiting Professor at Prometheus, KU Leuven. He obtained his PhD at Department of Biochemical Engineering, University College London funded by the National Scholarship Foundation of Greece. Subsequently he joined KU Leuven where he obtained funding to initiate an autonomous research track and is currently ATMP Research coordinator, within Prometheus – the KU Leuven division of skeletal tissue Engineering. He is active in addressing bioprocessing/translational challenges that would allow scalable and robust manufacturing of adult progenitor/stem cells, required for moving ATMPs for skeletal healing into the clinic. His task is to promote interfaces with industrial partners aiming at valorising research solutions generated within the platform along the entire ATMP bioprocess pipeline and has initiated several collaborations at the academia/industry interface. His research aims are to: Design and engineer progressively complex yet autonomous skeletal 3D living implants adopting breakthroughs in organoid technologies (Tissue engineered ATMPs); Develop a panel of metrics to enable quantitative definition of biologic events ensuring activation and maintenance of mechanism of action; Link therapeutic functionality upon implantation to the patient to in vitro quality attributes of skeletal ATMPs; Develop automated devices that can contribute to a cell therapy industry 4.0 equivalent. Integrate in silico derived tools for a self-regulated/adaptive manufacture of next generation living implants.

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Emerging tools and needs for AAV vector characterisation

Interview

Fabien Dorange

2020 Vision


VECTOR CHANNEL: ASSAYS & TITERING


Fabien Dorange holds a PhD in virology and worked as a postdoctoral researcher in the gene therapy field . Before joining Genethon, Fabien was Head of R&D viral safety in a CRO company (Texcell, France). He joined Genethon in 2016 and currently heads the analytical development department. He is responsible for developing analytical assays for characterization and release testing of gene therapy AAV vectors.

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Regulatory insights and lessons learned for a new decade in cell & gene therapy

Spotlight Article

Interview

Joyce L Frey-Vasconcells

2020 Vision

Joyce L Frey-Vasconcells PhD is considered one of the foremost regulatory experts regarding cell therapies, combination products, gene therapies, tumor vaccines, and tissues and brings extensive regulatory expertise and experience for this unique group of products. Prior to starting Frey-Vasconcells Consulting, Dr. Frey-Vasconcells served 6 years as a regulatory consultant for Pharmanet. Prior to joining Pharmanet, she served more than 12 years at the FDA as the Deputy Director, Office of Cellular, Tissue, and Gene Therapies (OCTGT) with the Center for Biologics Evaluation and Research (CBER). She was instrumental in developing many of CBER’s science and public health policies regarding the regulation of cells, tissues, gene therapies, tumor vaccines, and combination products (tissue engineered products). In 2001, Dr. Frey-Vasconcells was named the Regulatory Expert for Cell Therapies at FDA. Since starting Frey-Vasconcells Consulting, Dr. Frey-Vasconcells has continued working with industry on an individual basis and with organizations whose mission is to foster product development in these unique areas of medical science. She brings extensive regulatory expertise and experience for this unique group of products.

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Perspective

Clinical development of ATMPs: hospitals as an exemption?

Spotlight Article

Perspective

Martin Hildebrandt

2020 Vision

The ATMP landscape continues to be characterized by the fact that academia represents the major source of innovation. Undoubtedly, academic institutions do not possess an industry-like capacity to vigorously pursue the full developmental pathway to market authorization. At the same time, Industry has brought the first products to marketing authorization and defined novel modes of interaction with academia regarding the procurement of starting materials, manufacturing steps including storage of the product, clinical application and performance of trials. With ATMP development set to continue attracting and also challenging clinicians and scientists, this opinion papers aims to discuss logistical, financial and regulatory issues that might help to reshape the academic environment and to resuscitate some concepts that may have contributed to the original idea of the Hospital Exemption Clause.

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Vision 2020: what could come about in the next decade for biotech?

Spotlight Article

Perspective

Michael Yee

2020 Vision

  More than 2,000 years ago, around 400 BC, Hippocrates revolutionized medicine by describing diseases for the first time in history. In fact, he is credited as the first to believe disease was caused naturally, not by the Gods. Beyond characterizing diseases, he is also credited with establishing the earliest forms of diagnosing diseases, including […]

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New frontiers: cellular immunotherapy beyond cancer

Spotlight Article

Perspective

Matthew Brook, Joanna Hester & Fadi Issa

2020 Vision

Cell-based immunotherapies have the potential to revolutionize our approach to patient care. The first steps towards routine administration of cell therapies are being taken with early clinical trials underway in transplantation and in patients living with autoimmune conditions such as Type 1 diabetes and inflammatory bowel disease. Here, we consider the need for cellular therapy in the clinical setting; the current position, with a specific focus on transplantation where significant steps forward are being taken, and what the next decade may hold in this rapidly developing field.

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Expert Insight

Cell and tissue starting materials for ATMPs

Expert Insight

Neil W McGowan, John DM Campbell & Joanne C Mountford

2020 Vision


Supply Chain Focus: Materials Collection & Logistics at the Clinical Point of Care


The recent, rapid expansion in the use of cellular therapeutics has vastly increased the need to collect and process suitable starting materials from cell or tissue samples. These materials vary from relatively simple peripheral blood cell populations to the more complex tissue materials or established cell lines or isolates. In an evolving regulatory environment, adhering to good practice for acquisition, transport and processing is increasingly complex. Also, to understand the outcome of variations introduced during the manufacturing process and to permit more efficient process development, extensive analysis and retention of samples of the starting material is essential. However, these critical early steps are often overlooked and not optimized or standardized. In this article, we address some of the main challenges for the supply of high quality, consistent starting materials including: transport and hold time, cryopreservation, initial processing and the analysis of tissue and cellular materials. Variation in current practise highlights opportunities to standardize handling and testing of some common starting materials in order to increase consistency and quality control during the manufacture of these technically challenging advanced therapy medicinal products.

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Challenges in the procurement of starting materials for ATMP manufacture

Expert Insight

Sharon Zahra, Lynn Manson, Lisa Jarvis & Marc Turner

2020 Vision


Supply Chain Focus: Materials Collection & Logistics at the Clinical Point of Care


Somatic cell, genetically modified and tissue engineered advanced therapies share a common generic supply chain with other substances of human origin such as blood components, tissues, minimally manipulated cells and solid organs. The quality, safety and efficacy of the medicinal product at the point of administration to the patient is contingent on the entirety of its provenance including the selection and screening of the donor, procurement and distribution of the starting material, manufacturing, and return to, and management within, the healthcare environment. Information, chain of identity and regulatory compliance must flow bi-directionally, often across several different organizational boundaries. Moreover, the heterogeneity of advanced therapies is accompanied by considerable diversity in supply chains and complexity and disruption to existing modes of operation which act as a barrier to adoption. Overcoming these challenges and realizing the benefits of this new generation of therapies requires a new philosophy of long-term collaboration, mutual understanding and partnership between commercial and healthcare organizations.

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Performance of in-house optimized orthogonal methods for titration of in-process and purified rAAV

Expert Insight

Franz Schnetzinger, Kamila Pytel & Helena Pudilova

2020 Vision


VECTOR CHANNEL: ASSAYS & TITERING


To be able to accurately characterize gene therapy products it is crucial to develop robust analytical strategies. For this we developed and optimized methods for quantification of rAAV genome (vg/mL) and capsid (vp/mL) titers, measured by qPCR/ddPCR and rAAV capsid ELISA assays, respectively. Our focus was on optimizing sample preparation steps and sample pre-dilution conditions into standardized protocols to improve on the assay precision and robustness. Using orthogonal methods, we confirmed the optimized conditions and found ddPCR to have superior performance over qPCR; particularly for in-process material. Overall our data emphasize the importance of thorough optimization and standardization of in-house developed analytical methods for advanced characterization of purified and in-process derived rAAV productions. Furthermore, we have established HPLC based protocols which correlate with viral titers observed by qPCR/ddPCR and ELISA and hence can significantly improve throughput and sample turnaround time.

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