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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Developing automated cell processing strategies with Olivier Waridel, GE

As previously the CEO of medical device company Biosafe and now GM of Cell Banking & Point of Care at GE Healthcare, Olivier Waridel understandably feels positive about the recent partnership between the two companies. For him, the collaboration broadens the possibilities for both companies, ultimately working towards a ‘full solution for players in the CAR-T and immunotherapy space’.

‘Automatising’ cell processing is one of the developments Waridel has worked on and is most proud of. Using the example of cord blood banking Waridel outlines the process development ‘all the way from a sample in a bag to the cyro-preservation tank…[we] developed the tools for the whole processing of that in the laboratory’.

Cord blood banking is just one such example, and Waridel goes on to explain his work in ‘the clinical trials going on in the regenerative medicine space where it is required to have a closed system and automation for orthopaedic, cardiovascular and other trials that are going on. Our instruments can automate the process in the operating room; getting the cells ready for the re-infusion into the patient.’

The cell therapy space is a natural progression for Biosafe, allowing them to build on their previous experience of automating the cell processing line. Waridel explains how they use the same technology they already employ, but are also introducing a new instrument, Sefia, which has been specifically developed for the cell therapy space. Sefia will assist them with ‘running through the different cell manipulations and will help with the delivery to the end patient’.

In terms of differentiating their product from similar instruments on the market, Waridel highlights that Biosafe’s is centrifugation based:

‘The beauty is the technology in itself, so it allows us to process volumes and the way the cells are centrifuged allows us to go into detail into which lap of cells we are collecting and offers a lot of flexibility.’

The technology also allows them to include customers within the process development, building increasingly fluid relationships. Waridel aims to increase ‘reliability and stability’, and therefore increase the number of patients they can treat.

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

How the commercialisation of gene and cell therapies can revolutionise modern medicine

‘If one or two CAR-T therapies get to market it will provide impetus and momentum behind the industry’ – Dr Akshay Peer, Vice President of Sales and Account Management at TrakCel discusses the revolutionary steps that cell and gene therapies are making through their increased commercialisation.

In a field that is constantly seeing developments, Dr Peer outlines CRISPR and CAR-T therapies as forms of gene editing that can dramatically change the face of gene therapies and their use within modern medicine. He believes that as the commercialisation of these treatments increases, the regularity of their use will as well.

This is due to the surge in ‘positive public opinion’ that will arise from the successful implementation of the therapies. Dr Peer identifies the positivity that already exists around the new therapies, with ‘everyone looking at when these therapies will come to market and how much they will cost’. Although not going into detail about the financial outcome, he outlines his hope that when the public can see the lives of adults and children enhanced by using these therapies, any uncertain opinions will change to ones of optimism and confidence.

Peer acknowledges the lack of information that can exist around the new therapies due to an unfamiliarity in their usage; ‘People can sometimes get ahead of themselves and not understand completely what we’re trying to do here in this industry’. However, he is sure that this will change once there is evidence of patients who are successfully treated.

There is clearly excitement developing around the use of gene therapies, outlined by Dr Peer.

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

Sourcing raw materials for cell therapy manufacture – Dieter Hauwaerts, Celyad

‘Start working on raw materials early on’ – for Dieter Hauwaerts, Vice President of Operations at Celyad, the way to combat one of the biggest challenges in cell therapy manufacture is clear.

Limited availability of raw materials is a major issue for large scale manufacture of new therapies, but planning well in advance can help mitigate it: ‘In process development, take into account the quantity and quality of raw materials you will need in a clinical stage or commercial manufacturing process and build good relationships with suppliers.’

However, even with the necessary raw materials, Hauwaerts still sees issues with ‘the way we can manufacture products in a reliable and consistent way’, though he hopes that in 2017 automation will begin to negate this.

Watch the full exclusive interview with Hauwaerts – filmed at Cell Therapy Manufacturing & Gene Therapy Congress 2016 – above or here.

[Whitepaper] Regulatory Perspectives and Considerations for Cell & Gene Therapies

An Exclusive Whitepaper

Whitepaper Overview:

Recent clinical trials in the field of cell and gene therapy demonstrate remarkable therapeutic benefits with excellent safety. Despite demonstrated therapeutic effects, the commercialization of cell and gene therapies and their patient outreach remain scarce. Much of the research and development on cell and gene therapies is performed either on an academic level or by small and medium enterprises, largely excluding large pharmaceutical companies. Regulatory approval for cell and gene therapeutic products is performed on an individual product basis and is classified based on the degree of manipulation and intended end use. The primary deterrents to the lackluster commercialization of cell and gene therapies include the inherent complexities of the cells, issues with scalability for manufacturing and logistics and the complex regulatory requirements and time-consuming clinical trials. Cell and gene therapy products also have to navigate through the disparities in the regulatory requirements across regions. Furthermore, the complexity of product classification, extra requirements for combination of cell and gene therapies with a medical device, extensive paperwork surrounding the often ambiguous certification procedures and most importantly, the lack of harmonization of regulations across regions deter new investments and innovation in the field.

Limited understanding of the complex interactions of cell and gene therapeutics, absence of established standards and relatively scarce research data on the mechanism of action of these therapeutics make it difficult for stakeholders to navigate the complex and stringent regulatory requirements.

We elaborate the fundamental regulatory concerns associated with the development of cell and gene therapy products, and the need for international harmonization of regulatory requirement for approval of cell and gene therapies. The paper also addresses specific regulatory aspects in the EU and Japan as well as the roll-out of fast track mechanisms for market authorization in the EU and Japan. Finally, the paper addresses the urgent unmet need to provide regulatory certainty in the field of cell and gene therapeutics in the fast evolving global regulatory landscape.

 

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Hacking The Cell

Computational systems as diverse as vending machines, computers, and cells have something in common: They are all finite-state machines. That is, they may have an initial state, accept inputs, pass to different states according to the inputs received, and even enter states that lead to specific outputs. This kind of activity has been exploited in cells, but not with the degree of control we take for granted with the humble vending machine, which can dispense the right snacks depending on whether we press, say, B4 or C2. The vending machine can even refuse to dispense anything until a certain threshold has been surpassed (the right combination of coins).

In hopes of installing this kind of control into cells, MIT scientists have developed a way to program cells to respond to up to three different inputs. Crucially, the programming is capable of recognizing the order of inputs and responding accordingly. Although three inputs permit just 16 states, the MIT researchers point out that their method is scalable. It could accommodate additional inputs and thereby enable many additional states, enough to permit complex processes such as disease progression and cell differentiation to be tracked. The new synthetic biology approach could even make it possible to intervene in these processes, leading to cancer therapies or guiding the outcome of stem cell development.

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Progress Toward Commercial Scale and Efficiency in Cell Therapy Bioprocessing [Whitepaper]

Whitepaper Summary:

Currently there are 672 cell and gene therapy companies worldwide and 20 products approved by the food and drug administration (FDA). Dendreon’s Provenge autologous cell therapy although approved by the FDA ultimately failed commercially due to a manufacturing and distribution model that was not efficient. Cost of Goods (CoGs), manufacturing process and logistics are critical to the success of cell therapy commercialisation and these need to be considered from the inception of a cell therapy company in addition to the clinical science. Three key enablers for success are manufacturing automation/ single use technologies, a diverse pipeline in modularised facilities, and sophisticated data acquisition/ logistics.

Quality by Design (QbD) is a scientific, risk-assessment framework for process design based on relating product and process attributes to product quality. A risk assessment is conducted to prioritize the study of the most influential critical process parameters and material attributes. In addition to reducing risk QbD also increases efficiency as critical experiments are front loaded (Figure 1). This is particularly important in identifying required changes to the manufacturing protocol early reducing comparability risks.

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