The ‘transformative power’ of gene editing, with David Sourdive, Cellectis

‘Gene editing has a very transformative power in adaptive T cell therapy. In gene editing, we can make T cells do much more than they would normally do without being engineered.’ In this far-ranging interview, David Sourdive, Executive Vice President of Corporate Development at Cellectis, outlines the exciting updates from Cellectis’ current CAR-T therapy projects, from manufacturing, to clinical trials. He details his hope for the future and highlights the challenges that working with T cells can bring.

The possibilities of using gene editing to leverage the power of T cells into ‘better killers’ to ‘overcome the defence mechanisms of tumour cells’ are clear for Sourdive. Whilst acknowledging the current successful use of autologous treatments, he believes the industry is moving towards the possibility of ‘real pharmaceutical off the shelf T cell products’. There is an undeniable enthusiasm from Sourdive about gene editing to ‘maximise the power of T cells’, which can develop treatments that will meet the standard of care that chemotherapies and antibody therapies have.

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The importance of creative solutions in cell therapies

Pim Hermans, Director of Ligand Discovery at Thermo Fisher Scientifics, emphasises the importance of the symbiotic relationship Thermo Fisher has with their customers. For Hermans, this was key for their breakthrough product – ‘affinity products…for purification of biotherapeutics’ – which has been ‘really adopted by the industry’. It is widely used in the manufacturing of medicines, ‘especially in the gene and cell therapy field’. His example is the current use of AAV, which is now being used in ‘manufacturing at a large scale’.

By seeing customers as partners, Hermans’ philosophy is that ‘together we can make our products to suit the needs of their molecules’. He is confident in their ability to produce intuitive products and ‘do the best we can to provide the solutions’. He acknowledges ‘it can be challenging, but we are there to face those challenges together to try to solve problems to make good products’. This has been paramount to the evolution of cell and gene therapies.

Speaking of the new projects Thermo Fisher are currently working on, Hermans talks of the ‘next stage’ that will result from gene therapy evolution. He highlights the lentivirus as a new challenge, due to its nature as a more demanding viral vector, but a challenge he is eager to embrace.

Watch the full interview, filmed at Cell Therapy Manufacturing & Gene Therapy Congress, with Pim Hermans above or here.

‘The emerging technology revolution that is our cell and gene therapy space’

The gene editing tools and strategies for successfully taking a gene therapy to market.

Michael Mendicino, Owner, Chief Consultant & Advisor at Hybrid Concepts International, is excited to ‘be part of the emerging technology revolution that is our cell and gene therapy space’. We caught up with him at Cell Therapy Manufacturing & Gene Therapy Congress to discuss the current trends in gene therapies.

For Mendicino, the improvement in gene editing technologies is one of the most important recent developments in the industry. Although not necessarily a new or unfamiliar concept, he emphasises that the new technologies that are continuously emerging, such as Zinc Finger, TALENs and CRISPR, are vital to the future of gene therapies.

Mendicino sees a number of potential breakthroughs that are currently in the process of development that will allow for these new technologies to become viable treatment options:

‘For 2017 everyone is looking to see a licenser for marketing authorisation for the CAR-T product either coming from Novartis or from Kite Pharma. I think that will be tremendous for the field and will legitimise the field, not only for the companies that apply for the BLA (Biologics License Application) and get approval , but also for other companies that are not that far behind them, specifically in the CAR-T space, but also in cell and gene related product spaces.’

When building a BLA strategy that meets all the requirements, Mendicino breaks it down into three disciplines that need to be considered separately, as well as in conjunction with each other. Firstly, there is the ‘chemistry, manufacturing or controls’, or the ‘quality’ of the product. The second is ‘pharmacology and toxicology’, also known as the ‘pre-clinical or the non-clinical’, and the third is the clinical. Often these three considerations are handled in parallel, but this may need revising throughout the process. This is due to factors such as the product itself, as well as ‘the clinical indications sought, the patient population, the recruitment rates and whether or not the company has gone through some major CMC change that required scale up for commercial inventory.’

Watch the full interview as Michael Mendicino explores the methods of helping gene therapies progress to market above or here.

Proving the efficacy of cell therapy treatments to convince investors

‘What convinced investors, and what convinced our alliance partners were the clinical result we had in chronic lymphatic leukaemia, I think that’s important. I think with animal models, they are very helpful in a sense, in just the bare bones test system, but to prove efficacy, in the end is to run clinical trials.’ Jan Joseph Melenhorst, Director of Product Development & Correlative Sciences Laboratory from the University of Pennsylvania, discusses the necessary steps that must be taken for their cell therapy to become a viable treatment option.

Within his department at the university, Melenhorst examines the post infusion specimens of cells to better understand the potency and toxicity of the cells they infuse into patients. To enhance the potency of cells, additional gene editing tools can be included to modify the genome of the T-Cells and further enhance the expression of the targeting gene. Melenhorst speaks of his own experience with the CRISPR-Cas9 gene editing tool, but also draws upon the use of TALENs, stressing its importance to ‘the future of autologous and allogeneic cell therapies’. It is the autologous therapies Melenhorst believes are the current future of CAR-T therapy. Continue reading “Proving the efficacy of cell therapy treatments to convince investors”

Affordable Breakthrough Therapies

An Interview with Denis Bedoret of MaSTherCell

We recently sat down with Denis Bedoret, Chief BD Officier at MaSTherCell in Amsterdam at the Cell Therapy Manufacturing & Gene Therapy Congress conference to discuss the critical issues and opportunities present in the cell & gene therapy industry today.

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Novel Strategies for Gene and Cell Therapies

By Manuel J. T. Carrondo, Prof. Chem & Biochem Eng., FCT/UNL & Vice-president, iBET

Since the early nineties iBET has been involved in production and purification of viruses for gene therapy. Early on, enveloped retroviruses and non-enveloped adenoviruses where the targets; from late nineties onward lentivirus and baculovirus were added to the portfolio of enveloped viruses and AAV to the non enveloped viruses.

Although originally meant for monogenetic diseases, now some are also produced for cancer treatment (ex. oncolytic adenoviruses made in A547 cells, so replicative) or as reagents for cell therapies (also known as ex-vivo gene therapies).

Having developed scaled down tests and analyticals (including surface plasmom resonance, dynamic light scattering) coupled with its chemical engineering model competencies, iBET has designed membrane or media materials for which our key partners MERCK Millipore, SARTORIUS, GE HealthCare have developed the prototypes tested on our biologies and equipments. In this way, improved DSP processes have been created increasing viral yields and infective to total particle ratios or yields and viability for cell therapies.

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Cell Therapy Manufacturing and Gene Therapy Digital Week

27-30 March, 2017

KNect365 is pleased to introduce a special Digital Week program, comprised of a week-long series of free webinars with live Q&A – connecting cell and gene therapy leaders throughout the year from the comfort of your desk.

Cell Therapy Manufacturing & Gene Therapy Digital Week connects cell and gene therapy leaders to drive manufacturing and commercialisation through direct access to innovative discovery, product development, and regulatory know-how.

Register now to watch free educational sessions presented by leading industry experts, get answers to your toughest questions, network with colleagues and partners, and download useful resources.

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