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.
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.
For a recent KNect365 webinar, Christopher Bravery from Advanced Biologicals discussed the challenges of comparability for the ‘more complex’ biological molecules. He went into a detailed examination of purification, characterisation and activation, and their position within the space of biological comparability. Here we summarise the in-depth discussion that offers guidance and advice for those looking to meet the requirements of comparability for their molecules.
LISTEN TO THE WEBINAR HERE
First, Bravery outlines the differences between small molecules and biologicals which makes the characterisation of them a greater challenge for pharmaceutical companies. For small molecules, it is possible to know the exact molecular structure. When a cell substrate is introduced, the ‘manufacturing process is less clear’ as the ‘windows of characterisation are fogged with variability.’ This makes comparability much more challenging for cell based products as you are unable to define the molecular structure as easily. The proteins and the cell itself all vary in their phenotype, and are often more heterogenous, which adds to the uncertainty in the measurements taken.
Bravery defines comparability for biologics as the ‘need for a change in the process’ when the development is incomplete, either at post-approval or late stage. At this point, you should have characterised the raw materials to ‘understand critical quality attributes to establish process parameters’. He uses a hypothetical case study for a CAR-T product to demonstrate where comparability can need to be used within a development system, highlighting the area of ‘activation.’ (see diagram top)
Continue reading “Comparability for advanced therapy medicinal products with Christopher Bravery”
by Nick Hutchinson
Last month, speakers from UCB Pharma, Biogen Inc and Amgen were describing next generation processes development – platforms, products and plants during the opening plenary session of the BioProcess International European Summit. The industry, it would appear, is continuing to evolve its approach to manufacturing operations in response to changing market conditions and innovations in production technologies.
In the past, monoclonal antibodies typically made up a significant proportion of company’s pipelines. Recently, however, these pipelines have become more diverse and so the manufacturing networks of large biopharma companies need to be “modality agnostic”. This diversity extends beyond just the type of molecule being produced but also extends into the dosing regime, such that drug product facilities must be suitably adaptable.
Increasing drug potencies and specificities are leading to a downwards trend in the production volume requirements for a given product. However, at the same time, products are nowadays expected to be supplied to global markets rather than a handful of geographical regions. Uncertainly in forecasted demand is growing as firms extend their reach into previously unchartered markets and the industry become more competitive. Biopharmaceutical companies are placing greater emphasis on developing agile manufacturing networks and larger firms see operations functions as a key value driver to be integrated into corporate goals.
Continue reading “Biomanufacturing networks and the next generations of production facilities”
‘It’s amazing to think about the opportunity we create with other colleagues in the CAR-T field where we see patients who have no options – refractory tumour patients where a doctor tells them we cannot do anything else for you – we can come in with a new therapy.’ At Cell Therapy Manufacturing & Gene Therapy Congress we sat down with Markwin Velders, Vice President of Operations of Kite Pharma, to discuss the use of CAR-T therapies as a revolutionary treatment for populations of patients who were previously untreatable.
Vedlers is excited by recent breakthroughs in the use of CAR-T therapies in cancer patients and confident that they’ll continue. He emphasises patient populations, such as those with non-Hodgkinson Lymphoma, that can ‘now be treated effectively and can even reach 60-70% remission’, a group ‘who never had that option before’. He describes it as ‘an unmet medical need where we make a difference’, which offers a hopeful insight into the progress Kite Pharma are currently making in the field.
Kite has had success using immunotherapies to treat haematological, or liquid tumours, but Velders feel that it is in the much ‘wider field’ of solid tumours that they should be looking to make progress on current therapies. Solid tumours make up the largest number of cancer sufferers, and Velders hopes to ‘embark on the next phases’ for treatment ‘where there are real chances for the therapy as well as other opportunities’.
Continue reading “70% remission in previously untreatable cancers: CAR-T therapies with Markwin Velders, Kite Pharma”
‘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”
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