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

Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Issue Vol 2 Issue 3

Foreword

Advanced cell therapies – progress towards the clinic

Spotlight Article

Foreword

Chris Mason & Elisa Manzotti

Advanced Cell Therapies – Progress Towards the Clinic

The emergence of cell-based therapies has been one of the most remarkable advances in translational science over the last decade. The current spotlight discusses some of the emerging technologies and challenges in advancing cell-based therapies to the clinic.

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Editorial

Recent advances in the purification of recombinant adeno-associated virus

Spotlight Article

Editorial

Rachid Benchaouir

Advanced Cell Therapies – Progress Towards the Clinic

Catherine Greene Headshot

Recombinant adeno-associated viruses (rAAV) serve as powerful tools in research to establish and/or validate gene transfer protocols and as gene therapy vectors in pre-clinical developments and clinical trials. Its recent application in the context of the CRISPR-Cas9 genome editing strategies could predict a short-term therapeutic revolution. These remarkable achievements are the result of many biotechnological innovations that process rAAV vectors and produce large quantities of high-quality vector stocks. This editorial highlights the recent advances in the purification of rAAV vectors and discuss the prospects to open this gene therapy approach to future innovative and promising clinical trials.

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Commercial Insights

Cell & Gene Therapy Commercial Insight – September 2016

Spotlight Article

Commercial Insights

Mark Curtis & Richard Philipson

Advanced Cell Therapies – Progress Towards the Clinic

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.

CELL THERAPY:

Industry and investors alike have placed their bets on autologous T-cell immunotherapies as the future of oncology. Some have deviated from the pack and made advances on the allogeneic side, namely Cellectis, which is developing gene-edited donor-derived T cells that evade immune detection. Although progress with directed differentiation of pluripotent cells to T cells is a threat that could disrupt the current trajectory. Fate Therapeutics announced a deal with Memorial Sloan Kettering Cancer Center (MSKCC) this past month that will focus on the development of a truly scalable platform for the production of engineered T cells from pluripotent sources. While directed differentiation has a complex IP space, Fate will lay exclusive claim to patents recently generated at Sloan Kettering for the production of both T and NK cells.

GENE THERAPY:

This month sees exciting developments in the clinical arena, with Bluebird Bio announcing the initiation of a Phase 3 clinical trial in transfusion-dependent beta-thalassemia, and Alnylam announcing positive initial data from its Phase 1 study in healthy volunteers, investigating its RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1. Alnylam has a rich pipeline of RNAi therapies and, with its announcement of plans for a European hub in the UK, is clearly confident in its future value and success. Balancing this positive news is the announcement of the disbandment of Novartis’s cell and gene therapy unit. Although the company has committed to continuing to develop CAR-T therapies, this can only be seen as a blow to the field, and is reflected in the negative effects on the valuations of fellow CAR-T players Kite and Juno.

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Clinical Trial Insights

Clinical Trial Insight: Cell and Gene therapy Q3

Spotlight Article

Clinical Trial Insights

Alexey Bersenev

Advanced Cell Therapies – Progress Towards the Clinic

Dr Alexey Bersenev, Yale University, USA, provides an expert overview of the most important clinical trials, cases and cohort studies conducted in academic and industry with particular focus on later-stage efficacy data.

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Interview

Strimvelis: learnings from a cell and gene therapy forerunner

Spotlight Article

Interview

Dr Sven Kili

Advanced Cell Therapies – Progress Towards the Clinic

Dr Sven Kili is the VP and Development Head for the Cell and Gene Therapy division of GSK where he leads the teams developing ex-vivo Gene Therapies for a variety of genetic disorders. They are currently developing solutions for a number of diseases including ADA-SCID; WAS; Metachromatic Leukodystrophy (MLD) and Beta-Thalassemia. Prior to this, he was Senior Director, Cell Therapy and Regenerative Medicine for Sanofi (Genzyme) Biosurgery where he led the clinical development and medical affairs activities culminating in the granting of the first combined ATMP approval in the EU for MACI®. His team also prepared and submitted Advanced Therapy regulatory filings for Australia and the US, including health technology assessments and he was responsible for late stage developments for Carticel® and Epicel® in the US. Before joining Genzyme, Sven worked for Geistlich Pharma where, in addition to leading the cell therapy medical activities, he oversaw all UK regulatory functions and was the QPPV for the EU. Sven trained as an Orthopedic surgeon in the UK and South Africa and since leaving full-time clinical practise has developed expertise Cell and Gene Therapy in clinical development, regulatory compliance, value creation, risk management and product safety, product launches and post-marketing activities. He sits on the board of a Swedish Stem Cell company and still maintains his clinical skills in the UK NHS and serves as an ATLS Instructor in his spare time.

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Translation of cell therapies for neurodegenerative diseases

Spotlight Article

Interview

Prof. Olle Lindvall

Advanced Cell Therapies – Progress Towards the Clinic

Olle Lindvall received his PhD in 1974 and MD in 1978 and became Professor and Senior Consultant in clinical neurology at Lund University Hospital, Sweden, in 1992. He is Senior Professor since 2014. He was leading the clinical cell transplantation program for Parkinson’s patients at Lund University Hospital between 1983 and 2012. This program pioneered the use of neuronal replacement as a novel therapeutic strategy to restore function in the diseased human brain. Lindvall’s experimental laboratory is working with transplantation of stem cells and reprogrammed cells, and neurogenesis from the brain’s own neural stem cells after various insults. Olle Lindvall has published around 500 articles, review articles and book chapters. In 2007-2008, he Co-chaired the International Society for Stem Cell Research Task Force for the Clinical Translation of Stem Cells. Lindvall has received numerous Prizes and Awards. Since 2010, he is the Chairman for the Class for Medical Sciences at the Royal Swedish Academy of Sciences.

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Expert Insight

The route to patient access: challenges of evaluating & funding new cell & gene therapies

Expert Insight

Deborah Morrison & Nick Crabb

Advanced Cell Therapies – Progress Towards the Clinic

Almost every patient or parent, or carer of a patient with a long-term, life-threatening or seriously debilitating condition dreams of the day when medical science can provide a ‘cure’ for that condition. But what is the impact on patients and the healthcare system when it looks increasingly likely that the dream may be realized but […]

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In vitro red cell manufacture for clinical application

Spotlight Article

Expert Insight

Joanne C Mountford, Robert J Thomas & Marc L Turner

Advanced Cell Therapies – Progress Towards the Clinic

Red cell transfusion is an important part of current medical practice with around 92 million units transfused per annum worldwide; however global provision is limited by factors including sufficiency of supply, immunological incompatibility and the risk of transfusion-transmitted infections. The production of red cells in vitro is therefore an attractive goal; however such endeavors face many challenges including those arising from the complex biology of erythropoiesis, the scale of production required – a single red cell unit contains approximately 2×1012 cells – and the new developments needed in both upstream and downstream processing. Many of these challenges are common to other next-generation cellular therapeutics and so development of in vitro red cell production may also inform more general approaches to large-scale cell manufacturing where the cell, rather than a protein or factor it produces, is the desired product. In this article we discuss the alternative strategies for production of red cells in vitro and the progress on the scientific and engineering challenges that need to be addressed to make manufactured red cells a clinical reality.

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Recent advances in conditional cell immortalization technology

Spotlight Article

Expert Insight

Ivan B Wall, Gerardo Santiago Toledo & Parmjit S Jat

Advanced Cell Therapies – Progress Towards the Clinic

Gene-modified cell therapies are transforming medicine. Over the last 18 months, notable clinical successes using antigen-targeting cellular immunotherapies have been achieved. However, another kind of gene modification has significant potential for the cell therapy industry. The development of fully controllable transgenes has enabled the creation of conditionally immortalized cells that can be expanded to clinical quantities in a stable and consistent fashion, yet can be returned to a normal, non-dividing state for therapeutic delivery to the patient. In this article, we discuss some of the key technologies that have been used to create conditionally immortalized cells for clinical development.

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Unlocking the therapeutic and commercial potential of CAR-T technology

Spotlight Article

Expert Insight

Michelle J Smith, Brittni M Peterson & Barbara A Nelsen

Advanced Cell Therapies – Progress Towards the Clinic

The striking clinical results of CAR-T therapies in blood cancers have shifted the cell therapy field from one that has future potential, to one offering hope of a future to those living with cancer. Since 2012 the field has expanded beyond investigators to investment and initial public offerings, individual ‘cures’ and industry growth. Here, we review the current state of CAR-T therapeutics, from improvements in CAR-T technology to clinical results, business growth and future applications. Finally, we discuss the remaining barriers to successful commercialization of CAR-T technology identified by key investors within this rapidly expanding field.

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Development of iPSC technology in Parkinson’s Disease

Spotlight Article

Expert Insight

Jun Takahashi

Advanced Cell Therapies – Progress Towards the Clinic

The central nervous system has very little potential for regeneration which is why cell replacement therapy offers great potential for functional recovery in neurodegenerative diseases. Following the discovery of human embryonic stem cells in 1998 and induced pluripotent stem cells in 2007, we are now able to manipulate the quantity and quality of donor cells utilized in stem cell-based therapies. For Parkinson’s disease in particular, which will be the focus of this article, precise protocols to establish induced pluripotent stem cells and to derive dopaminergic neurons have been developed to clinical grade, and preclinical data concerning the efficacy and safety of these cells now exists for rodent and monkey models. Based on these efforts, clinical trials for a number of neurodegenerative disease are expected to commence in the near term.

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Harnessing the power of cardiac stem cells

Spotlight Article

Expert Insight

Prof. Christine Mummery

Advanced Cell Therapies – Progress Towards the Clinic

Eric-Kunkel

Christine Mummery studied physics at the University of Nottingham, UK and has a PhD in Biophysics from the University of London. After holding positions including a postdoc and tenured group leader at the Hubrecht Institute, she became professor at the UMC Utrecht in 2002. In 2008, she became Professor of Developmental Biology at Leiden University Medical Centre in the Netherlands and head of the Department of Anatomy and Embryology. Her research concerns heart development and the differentiation of pluripotent human stem cells into the cardiac and vascular lineages and using these cells as disease models, for safety pharmacology, drug discovery and future cardiac repair. She is a member of the Royal Netherlands Academy of Science (KNAW), and board member of the International Society of Stem Cell research (ISSCR), the KNAW and the Netherlands Medical Research Council (ZonMW). She was recently awarded the Hugo van de Poelgeest Prize for Animal Alternatives in research. She co-authored a popular book on stem cells “Stem Cells: scientific facts and Fiction” in 2011 (2nd edition 2014) and is Editor-in-Chief of the ISSCR journal Stem Cell Reports.

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