Journal Archive

Commercial Insights

Cell & Gene Therapy Commercial Insight – December 2017

Mark Curtis & Richard Philipson

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:

Interest in pluripotent stem cell (PSC)-derived products remains strong and we continue to make ground on the creation of off-the-shelf designer cells. Fate Therapeutics, through its partnership with Sloan Kettering, has made some particularly interesting advancements. Recently, at the American Society for Hematology Annual Meeting in December, the company announced that it has successfully created T cells with multiple modifications executed through a single CRISPR/Cas9-mediated engineering event. The cells Fate created were derived from a clonal iPSC line that was modified to introduce a chimeric antigen receptor (CAR) and remove T-cell receptor (TCR) expression, giving them the ability to target cancer while effectively making them ‘invisible’ to the immune system. Fate also reported on the generation of iPSC-derived NK cells, which will be used for IND enabling work.

GENE THERAPY:

This month sees the outstanding achievement of Spark Therapeutics in the landmark approval by FDA of LUXTERNA™ for the treatment of RPE65 mutation-associated retinal dystrophy – the first FDA-approved gene therapy for a genetic disease. December always sees a rash of news from the American Society of Hematology annual meeting, and this year is no different. News from BioMarin on progress with its treatment for hemophilia A will certainly keep Spark on its toes, as it looks like BioMarin is ahead of its competitor in the race to be the first to market for this serious hereditary bleeding condition. A third key player in the hematology field, bluebird bio, also keeps up the positive news flow with updates suggesting favorable clinical outcomes in patients with sickle cell disease or beta-thalassemia receiving treatment with its lentivirus-based therapy. All in all, a great way to end the year!
DOI: 10.18609/cgti.2018.002
Citation: Cell Gene Therapy Insights 2018; 4(1), 9-21.

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Cell & Gene Therapy Commercial Insight – January 2018

Mark Curtis & Richard Philipson

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:

We’ve seen a flurry of M&A activity the last several months, which began with the acquisition of Kite by Gilead in August of 2017 for $11.9 billion. This was followed by the bolt-on acquisition of Cell Design Labs for $566 million, also by Gilead, in December 2017. We kicked off this year with the announcement that Takeda will purchase TiGenix for $620 million to get access to global rights to Cx601. In a response to the Kite-Gilead deal, Celgene swooped in to buy Juno for $9 million this past month, the second major acquisition we’ve seen in 6 months in the T-cell space. Perhaps not surprisingly so. Pipelines are maturing and technology is beginning to be validated in the blood cancer space, with not one, but two approvals in recent times, including Kymriah and Yescarta. The acquisitions of Juno and Kite leave Adaptimmune as the last major independent T-cell biotech in the USA.

GENE THERAPY:

The start of the year sees signs of progress in a couple of company’s pipelines, with GenSight getting the nod from MHRA to start its Phase 1/2 study in retinitis pigmentosa and Ultragenyx releasing positive news from the first cohort of patients with OTC deficiency treated with its AAV8-based vector in the PIONEER Phase 1/2 clinical trial. Interesting also to see the tie-up between Synpromics – an Edinburgh-based company focused on synthetic promoters – and Professor Adrian Thrasher’s clinical research group at UCL Great Ormond Street Institute of Child Health. Thrasher’s group has a well-established reputation for excellence in gene therapy for rare immunodeficiencies, so it will be worth watching for what emerges from this new collaboration.

DOI: 10.18609/cgti.2018.008
Citation: Cell Gene Therapy Insights 2018; 4(1), 57-66.

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Clinical Trial Insights


Expert Insight

Fueling a Commercial Reality: Optimization of Separation & Expansion Across the Manufacturing Pathway

Spotlight Article

Interview

David DiGiusto

Separation and Expansion Technologies

Chris Wiwi

Dr David DiGiusto is the Executive Director of Stem Cell and Cellular Therapeutic Operations for Stanford Hospital and Clinics and a Senior Academic Researcher in the Division of Stem Cell Transplantation and Regenerative Medicine at Stanford University. He has over 25 years of experience in the scientific, clinical and regulatory aspects of cells as therapeutic agents including the isolation, characterization and genetic modification of hematopoietic stem cells and T-cells for clinical applications. He has been instrumental in the creation of 6 GMP compliant biologics manufacturing facilities and associated quality systems, production and QC testing programs. Under his direction, plasmid DNA, CAR-T-cells, regulatory T-cells, engineered stem cell grafts and gene modified hematopoietic stem cell products have been manufactured and released for use in Phase I/II clinical trials. Dr DiGiusto is a major contributor to first in human (and other ongoing) studies for Cancer and HIV Gene Therapy and has developed methods for assessing ex-vivo stem cell manipulations using in vitro and in vivo models. His laboratory (The Laboratory for Cell and Gene Medicine) specializes in the development of manufacturing processes and QC assays and provides cGMP compliant clinical materials production and regulatory support activities for investigational cell products.

DOI: 10.18609/cgti.2018.001
Citation: Cell Gene Therapy Insights 2018; 4(1), 1-8.
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Technological developments in dielectrophoresis and its path to commercialization

Spotlight Article

Expert Insight

Michael Pycraft Hughes

Separation and Expansion Technologies

One of the bottlenecks for cell therapy development is the need to isolate specific cells, be it stem cells with specific differentiation fates, or specific white cells from a blood cell sort. However, the nature of the application means that the separation method should ideally be label-free and GMP-compliant, as well as achieving appropriate levels of throughput and cell recovery. One emergent field in cell separation is dielectrophoresis, an electrostatic method that has the potential to meet this growing need. Recent commercial developments mean that for the first time, this technique will be more widely available to the cell therapy sector.

Submitted for review: Jan 12 2018 Published: Feb 28 2018
DOI: 10.18609/cgti.2018.010
Citation: Cell Gene Therapy Insights 2018; 4(1), 81-88.
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Planning for Commercial-Scale Production: Tools & Technologies of the Future

Spotlight Article

Interview

David James

Separation and Expansion Technologies

 

David is an experienced international Executive who has spent nearly 30 years commercializing innovative technologies. His broad industry experience and balanced technical and commercial perspective have helped launch several market leading instruments, consumables and manufacturing systems for Cell Therapy and Diagnostics. David is a regular conference speaker, guest lecturer for the Department of Chemical and Bio-molecular Engineering (University of Melbourne) and author of multiple papers and articles.

DOI: 10.18609/cgti.2018.003
Citation: Cell Gene Therapy Insights 2018; 4(1), 23-29.
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Advances in Expansion Technologies for the Clinical-Scale Production of MSCs

Spotlight Article

Interview

Steve Oh

Separation and Expansion Technologies

 

Steve Oh is the Director of Stem Cell Bioprocessing and Institute, Professor/Scientist of the Bioprocessing Technology Institute (BTI) and an expert in integrated stem cell bioprocessing for the manufacture of human pluripotent/embryonic and adult mesenchymal stem cells (MSCs) for cell therapies. Key recent achievements include high density production of pluripotent stem cells, cardiomyocytes, neuroprogenitors and red blood cells in suspension bioreactors. In the MSC area, methods of producing high-quality MSCs, and primed towards cartilage and bone repair have been achieved. Recently a new assay for stem cell senescence useful for the quality control of stem cells for bioprocesses has been developed by his team. He has over 100 publications and 25 patents in stem cells production. He was the International Society of Cellular Therapies (ISCT) Co-Vice President (Asia) from 2015 to 2017, and led the planning of the Sessions for Commercialisation of Cell Therapies for the Silver Jubilee (25 years) ISCT meeting at Suntec City, Singapore in 2016. He is also the current Vice President of the Singapore Stem Cell Society and holds an Adjunct Professorship at the Nanyang Technological University. Steve has founded two stem cell companies, Brilliant Research and Veristem, and is a veteran of 26 years in the biotech industry. Brilliant Research is a company specializing in providing Personalised Stem Cell Banking and Stem Cell Bioprocess solutions to build the 3rd Pillar of Medicine, Cell & Gene Therapies.

DOI: 10.18609/cgti.2018.006
Citation: Cell Gene Therapy Insights 2018; 4(1), 45-49.
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Advances in Media Formulations for Clinical-Scale Expansion of Cells

Spotlight Article

Interview

Thierry Burnouf

Separation and Expansion Technologies

 

Prof Thierry Burnouf, PhD, is the Vice-Dean of the College of Biomedical Engineering, Director of the Graduate Institute of Biomedical Materials and Tissue Engineering, Director of the International PhD Program in Biomedical Engineering, and Professor in the International PhD Program for Cell Therapy and Regeneration medicine at Taipei Medical University, Taiwan. He has over 30 years of experience in translational research and development and industrial manufacture of human therapeutic plasma proteins (coagulation factors, immunoglobulins, alpha 1-antitrypsin, etc.) and cellular products, with a special interest in protein isolation procedures and virus inactivation and removal technologies. He has been closely involved into the drafting of WHO Guidelines on virus inactivation and removal procedures applied to human blood plasma products, production of animal-derived antivenoms immunoglobulin, and production, quality control and regulation of plasma for fractionation. He is a member of the working parties on Cellular Therapies and Global Blood Safety of the International Society of Blood Transfusion.

DOI: 10.18609/cgti.2018.005
Citation: Cell Gene Therapy Insights 2018; 4(1), 37-43.
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