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

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 6 Issue 1

Editorial

From laboratory to patients: dissecting obstacles in cell & gene therapy development

Spotlight Article

Editorial

Daniela Nascimento Silva, Carlotta Spano, Martina Varricchio, Giulia Grisendi, Milena Soares, Anna Pasetto & Massimo Dominici

Preclinical and translational R&D insights

    Looking at the cellular therapy landscape, there are more 34,000 clinical studies reported in https://clinicaltrials.gov (searching for ‘cellular therapy’ closed, past, active, enrolling, non-enrolling) [1]. While this number most probably over-estimates the clinical impact of cells as therapeutics, it is beyond doubt that cells are progressively entering into the clinical scenario for a […]

Read full article »
Commercial Insights

Cell & Gene Therapy Commercial Insight – January 2020

Commercial Insights

Mark Curtis & Richard Philipson

Preclinical and translational R&D insights

Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.

Read full article »

Cell & Gene Therapy Commercial Insight – December 2019

Commercial Insights

Mark Curtis & Richard Philipson

Preclinical and translational R&D insights

Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.

Read full article »
Interview

Key considerations for the use of suspension culture systems for viral vector manufacturing

Interview

Rénald Gilbert

Preclinical and translational R&D insights


VECTOR CHANNEL: SUSPENSION CULTURE SYSTEMS


Rénald Gilbert is a senior researcher at the Human Health Therapeutics Research Centre of National Research Council Canada (NRC) in Montreal. Throughout his career at the NRC, he has held various leadership roles including the Program leader of the Vaccines Program, which delivered on the Government of Canada’s mandate on innovation, fostering Small Medium Enterprise (SMEs) and public well-being. Currently as the lead of the NRC Biomanufacturing Research Initiative, he manages a portfolio of projects aimed at developing new and improved proprietary platforms enabling biomanufacturing of scalable, cost-effective and safe complex biological products. Dr Gilbert is also an adjunct professor in the department of Bioengineering at McGill University. His research interests include the optimization of viral vectors for the development of vaccines and for cell and gene therapy applications, as well as their methods of production. He is currently directing research projects aiming at increasing the yield and efficacy of vectors derived from adeno-associated virus (AAV) and lentivirus using suspension cultures of mammalian cells. Dr Gilbert completed his PhD in Biochemistry at McMaster University (Hamilton Ontario) in 1994. Before starting his career as a research officer at NRC in 2002, he got trained as a postdoctoral fellow in the department of Cell Biology of the Weill Cornell Medical College (New York City) and in the department of Neurology in Neurosurgery of McGill University.

Read full article »


Emerging upstream bioprocessing and analytical tools for vector manufacture

Interview

Michael J White

Preclinical and translational R&D insights


VECTOR CHANNEL: SUSPENSION CULTURE SYSTEMS


MICHAEL J WHITE is a Scientist in the BioProcess Development group at Ring Therapeutics. He has been working with/on viruses since 2012 as a postgraduate fellow at Yale University School of Medicine in Brett Lindenbachs’s Lab where he worked on virus assembly and viral protein interactions for Hepatitis C Virus. From there he went to Purdue University to pursue his doctorate in the laboratory of Richard Kuhn studying assembly and entry/infectivity mechanisms of flaviviruses such as Dengue and Zika, where alongside purifying virus, he developed expression platforms and purification processes for viral proteins. From here, he broke into industry and gene therapy as a postdoctoral fellow at Medimmune/AstraZeneca within the BioProcess Development Group. It was here that he worked on end to end bioprocess development of AAV vectors focusing on how upstream cultivation and harvest conditions from suspension cultures can influence downstream purification recoveries and vector potency. He is currently at Ring Therapeutics as team lead for upstream process development for a novel gene therapy vector.

Read full article »

Establishing a suspension culture-based lentiviral vector bioprocessing facility

Interview

Gabrielle Humphrey

Preclinical and translational R&D insights


VECTOR CHANNEL: SUSPENSION CULTURE SYSTEMS


GABRIELLE HUMPHREY, Associate Director is the lead of the Vector Manufacturing Science and Technology group at Adaptimmune, UK. In 2018, Gabrielle established the team responsible for starting-up Adaptimmune’s manufacturing capabilities at the Catapult CGT Manufacturing Centre in Stevenage. Her focus is currently on the production of GMP lentiviral vector for TCR T-cell therapy. Previously she has been involved in the GMP manufacturing of lentiviral vectors for CAR T-cell therapy and for gene therapy applications. Her area of expertise is the design and delivery of cleanroom bioprocesses for novel breakthrough therapies focusing on single-use disposable flow paths, aseptic processing and data monitoring.

Read full article »

Suspension systems in continuous viral vector production processes

Interview

Felipe Tapia

Preclinical and translational R&D insights


VECTOR CHANNEL: SUSPENSION CULTURE SYSTEMS


FELIPE TAPIA is a chemical engineer with a PhD in bioprocess engineering carried out at the Max Planck Institute in Germany. He specializes in continuous virus production and is a co-Founder of ContiVir, a startup project that develops fully continuous processes of gene therapy viral vectors. ContiVir received a €1.1 million grant from the European Union, the German government and the Max Planck Society and its development is taking place at the Bioprocess Engineering Group of the Max Planck Institute in Magdeburg (Director: Prof. Udo Reichl).

Read full article »

Streamlining preclinical-clinical translation processes for an expanding CGT pipeline

Spotlight Article

Interview

John Moscariello

Preclinical and translational R&D insights

JOHN MOSCARIELLO joined Celgene in 2018 and serves as the Senior Director of Viral Vector and Gene Editing Process Development. Prior to joining Celgene, John was the Vice President of Process Development at AGC Biologics (formerly CMC Biologics) where his organization was responsible for all cell line development, upstream and downstream process development, analytical and formulation development, and technology transfer and technical manufacturing support. Prior to AGC Biologics, John held director-level positions at Amgen, focused on process development and characterization, clinical and commercial technology transfer and process validation. John has a PhD in chemical and biological engineering from the University of Wisconsin-Madison and a bachelors of chemical engineering from the University of Delaware.

Read full article »

Tackling the plasmid supply bottleneck

Interview

Preclinical and translational R&D insights

MARCO FERRARI has a degree in Law at Luigi Bocconi University of Milan (Italy), with a specialization in Health Management at Imperial College Business School of London. Today Marco Ferrari is CEO of Anemocyte Srl, company of Nine Trees Group Spa, a private holding with five affiliated companies operating in the Life Science sector.

STEFANO BAILA received his PhD in 2007 based upon translational research and development of gene therapies for hemophilia at the Children’s Hospital of Philadelphia. Since that time he has been actively involved in the process development and manufacturing of advanced therapeutic medicinal products through business development and strategic marketing roles at Areta International, a CDMO, and by leading field implementation and commercialization activities for the cell processing unit of Terumo BCT. Stefano also worked as Industrialization Manager at Celyad where he led process development and automation efforts for CAR-T therapeutics. Now he serves as Director of Operation and Business Development for Anemocyte.

Read full article »

Programming superior cells: translational progress at the Altius Institute for Biomedical Sciences and Umoja Biopharma

Spotlight Article

Interview

Preclinical and translational R&D insights

SHON GREEN is trained at UC Berkeley and UCSF where she developed and employed preclinical models of cancer to study tumor development and potential therapeutic approaches. She utilized these expertise to drive the preclinical development of T cell-based immunotherapies targeting both solid and liquid tumors at Eureka Therapeutics Inc. (Emeryville, CA, USA), where she successfully guided two novel programs through preclinical proof-of-concept and safety studies to support investigational new drug applications and clinical testing. Since July 2018, she has been directing the development and clinical translation of novel genetic and epigenetic editing tools to enhance cellular therapies for cancer at Altius Institute for Biomedical Sciences (Seattle, WA, USA). She currently also serves as the head of Translational R&D at Umoja Biopharma, a new startup in Seattle, where she is leading the preclinical strategy to obtain proof-of-concept for potentially disruptive new approaches to cancer immunotherapy.

Read full article »

Analysing clinical trends for cell & gene therapy in neurodegenerative diseases

Interview

Roger Barker

Preclinical and translational R&D insights


CLINICAL TRENDS


ROGER BARKER is the Professor of Clinical Neuroscience at the University of Cambridge and Consultant Neurologist at the Addenbrooke’s Hospital Cambridge. He is a PI in the Wellcome – MRC Cambridge Stem Cell Institute and Director of the MRC funded UK Regenerative medicine hub working on Pluripotent Stem and Engineered cells. He trained at Oxford University and St Thomas Hospital Medical school and after completing his medical training took up an MRC Clinician Scientist Fellowship. For the last 25 years he has run research that seeks to better define the clinical heterogeneity of two common neurodegenerative disorders of the CNS- namely Parkinson’s (PD) and Huntington’s disease (HD). This has helped him define the best way by which to take new therapies into the clinic. In this respect he has been heavily involved in gene and cell based trials for patients with these conditions and currently co-ordinates an EU funded transplant programme using human fetal tissue for patients with PD, following on from an earlier MRC funded trial using similar tissue in HD. He is part of a new EU project (STEM-PD) and a global initiative (GFORCE-PD) that is seeking to take stem cells to trial in these disorders. He is currently is Co-Editor in Chief of the Journal of Neurology and sits on the editorial board of many other journals. He is on the research advisory board of the Cure PD Trust and Parkinson’s UK. In 2015 he was elected a Fellow of the Academy of Medical Sciences.

Read full article »

Building for future success: the Syncona Partners model

Interview

Martin Murphy

Preclinical and translational R&D insights


INVESTOR INSIGHTS


MARTIN MURPHY is Chief Executive Officer of Syncona Investment Management Ltd. He co-founded Syncona in 2012 with The Wellcome Trust. Since then, Martin has been closely involved in the foundation and development of five Syncona companies: Achilles, Autolus, Blue Earth, OMass and Quell Therapeutics. Previously, he was a partner at MVM Life Science Partners LLP, a venture capital company focused on life science and healthcare, where he led their European operations. Martin has also held roles with 3i Group plc and McKinsey & Company. He has a PhD in Biochemistry from the University of Cambridge.

Read full article »

State of the Industry: The Financial, Clinical, and Scientific Landscape for Cell and Gene Therapies

Interview

Janet Lynch Lambert

Preclinical and translational R&D insights


INVESTOR INSIGHTS


2019 was a year of significant growth in the regenerative medicine sector. Thousands of patients are now benefitting from commercial regenerative medicines, and the impact of the early cell and gene therapies is dramatic. The clinical pipeline is robust, with nearly a hundred Phase 3 trials underway, several late-stage products poised for approval, and next-gen technologies such as gene editing beginning to enter the clinic. Therapeutic developers are increasing their focus on solving manufacturing challenges. The sector continued to attract billions in investment, further fueling our scientific, clinical and commercial progress.
Each year, the Alliance for Regenerative Medicine (ARM), the international advocacy organization for the cell and gene therapy and broader regenerative medicine sector, hosts a Cell and Gene State of the Industry briefing, presenting global sector data at the Biotech Showcase at the JP Morgan Healthcare Conference. The industry overview is followed by two panels focusing on the latest developments in the sector. Recordings and slides are available on the ARM website [1]. This year, for our tenth annual briefing, I spoke to over 1,000 attendees in-person and online. This group of people – scientific and industry experts, regulators, payers, investors, and patient advocates – are incredibly knowledgeable and motivated to bring these therapies to patients across the globe, and we at ARM are grateful to be spearheading efforts to get safe and effective medicines to patients.
Since I came to ARM in 2017, this sector has really come of age – and there is still so much more to come. While 2019 was very successful, we still have considerable work to be do together to ensure that the early clinical promise and commercial successes of these therapies translates to widespread patient access. With that in mind, I wanted to share some of the major trends we’ve seen in 2019, as well as the outlook for 2020.

Read full article »
Perspective

Clinical Update: Breaking New Ground

Perspective

Sven Kili

Preclinical and translational R&D insights


CLINICAL TRENDS


Cell and Gene Therapy Insights proudly present the inaugural edition of Clinical Trends – a quarterly snapshot of key breakthroughs, challenges and areas of evolution in cell and gene therapy clinical development. Clinical Trends is specifically designed to provide an efficient means of updating yourself on the global trials landscape in general, whilst gaining more in depth insights into clinical progress in specific therapeutic areas and indications.

Read full article »

Complexity be damned: the need to better use biology to achieve more impactful cell therapies

Spotlight Article

Perspective

Nancy L Parenteau

Preclinical and translational R&D insights

Biological complexity is both a benefit and a bane of cell therapy. An astute partnership with nature is required to achieve a reliable clinical outcome from cell therapy. To accomplish it, we must dig deep enough to acquire knowledge crucial to translation, while maintaining a perspective that will prevent misdirection. Not every bit of information will be of equal importance, yet we can’t skip over or miss what could be pivotal. Dealing with the biological complexity surrounding cell therapy may seem like a risky balancing act. However, luck favors the prepared, and there are practices we can employ to reduce translational risk and form a clinically impactful collaboration between science and nature.

Read full article »
Expert Insight

Development and scale-up of suspension culture processes for viral vector manufacturing: challenges and considerations

Expert Insight

Helen Young & Philip Probert

Preclinical and translational R&D insights


VECTOR CHANNEL: SUSPENSION CULTURE SYSTEMS


Viral vectors are currently the preferred vehicle for delivery of DNA for cell and gene therapies, which have the potential to revolutionize the treatment of human diseases. Developing high yielding, robust, scalable and commercially viable processes to manufacture sufficient quantities of viral vectors that also meet the stringent standards of purity, identity, potency and safety presents challenges. This article will discuss some of these challenges and considerations for the development of viral vector production processes. We conclude that although the industry will see a variety of production technologies employed for the manufacture of viral vectors, suspension culture will become the predominant method of production due to its scalability and it likely being the only method able to generate sufficient quantities of product at commercial scale. We also anticipate that the industry will move away from using transient transfection-based processes and move towards stable producer lines in common with conventional biologics manufacture.

Read full article »

Manufacturing of human iPSC-derived cell therapies: road to the clinic

Spotlight Article

Expert Insight

Raquel Martín-Ibáñez & Dhruv Sareen

Preclinical and translational R&D insights

One of the major roadblocks in regenerative cell therapies is the lack of appropriate models of human diseases and the availability of relevant patient-specific cells capable of replacing damaged tissues and organs. Induced pluripotent stem cells (iPSCs) derived from patients provide a source of human tissue that addresses this problem and has the potential to create an entire new set of human disease models and ultimately cells for transplantation. However, coordinating the production of quality iPSCs at scale remains a challenge for most academic and industrial laboratories. In this article, we outline key clinical applications of human iPSCs, and challenges associated with at scale clinical manufacturing of iPSCs and their derivatives. Based on the experience of our team in producing, maintaining and distributing iPSC lines for a decade as a global provider of iPSC-derived cell solutions, the progress of the field in developing iPSCs and their derivatives for translating to the clinic has been reviewed here. The development of a scalable iPSC-based biomanufacturing platform will enable innovative and sustainable solutions for translating much-needed iPSC-derived cell therapies into the clinic.

Read full article »

The rise of human induced pluripotent stem cell technology in drug development

Spotlight Article

Expert Insight

Chun Liu & Joseph Wu

Preclinical and translational R&D insights

Drug development has been significantly hampered over the past few decades, partly due to our over-reliance on animal models and immortalized cell lines. Recent advances in human induced pluripotent stem cell (iPSC) technology provide an excellent physiologically relevant approach for disease modeling, compound screening, and preclinical evaluation of drugs. Human iPSCs generated from patients have unique advantages over conventional models, including the potential to differentiate into any cell type and still retain the genetic identity of the patients. The growing interest to adopt iPSC technology for drug development by researchers and pharmaceutical companies has become increasingly evident. Here we discuss in detail the recent advances in iPSC technology relevant to the process of drug development.

Read full article »

Vascular microphysiological systems to model diseases

Spotlight Article

Expert Insight

Qiao Zhang, Xu Zhang & George A Truskey

Preclinical and translational R&D insights

Human vascular microphysiological systems (MPS) represent promising 3D in vitro models of normal and diseased vascular tissue. These systems build upon advances in tissue engineering, microfluidics, and stem cell differentiation and replicate key functional units of organs and tissues. Vascular models have been developed for the microvasculature as well as medium-size arterioles. Key functions of the vascular system have been reproduced and stem cells offer the potential to model genetic diseases and population variation in genes that may increase individual risk for cardiovascular disease. Such systems can be used to evaluate new therapeutics options.

Read full article »
Twitter IconVisit Our Blog