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European Biopharmaceutical Review

Increasing the Accessibility of Life-Changing Treatments

Cell and gene therapies (C&GTs) offer hope for patients suffering from a range of cancers and rare diseases. This relatively new class of treatments has the potential to address the underlying causes of disease, compared to traditional approaches, which often address only symptoms. However, they are not readily affordable or, consequently, available to many patients.

There is a need to increase the accessibility of these therapies and there are multiple factors that will drive more widescale uptake. The manufacture of therapies and scaling approaches will reduce the cost burden associated with a manual process requiring highly trained individuals. Putting patients at the centre of this journey will not only keep them better informed of their treatment plans, but will also reduce the number of setbacks seen at the clinical trial stage, making the route to market easier. The development of these treatments can be complemented by new in vitro models that more accurately mimic in vivo environments, allowing more representative data to be collected at the early therapy stages. They might also identify a wider range of health conditions that could benefit from this type of treatment. Finally, understanding the commercial landscape – from regulatory pathways to supply chain logistics and reimbursement strategies – will facilitate more of these therapies moving from research to widescale use in the clinic.

Scaling the Manufacturing Process

To provide accessible treatments to a large patient group, the manufacturing process needs to be scaled seamlessly across the value chain from early-stage research, through process development, clinical trials, and into commercial production. There is an interesting contradiction within this scaling process that needs to be carefully managed. Towards the earlier stages of development, a large amount of flexibility is required as the appropriate culture protocols are established, whereas these protocols need to be locked down as the therapies transition to their manufacture at CMOs under GMP guidelines. This is the case for both allogenic therapies, where companies are looking to scaleup, and autologous therapies, where scale-out is necessary to produce ‘batch of one’ therapies.

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Hilary Thomas, healthcare and life sciences expert at PA Consulting, is a senior accredited clinician with academic, public sector, management, and consulting experience. She has been a keen clinical triallist, key opinion leader, and Professor of Oncology at the University of Surrey, UK. Hilary is motivated by having positive impact for patients, and in her consulting career, her team’s approach to transforming quality of care has benefitted patients in over 30 countries across numerous therapeutic areas. She is a former elected member of the General Medical Council and trustee of several healthcare charities. She has an extensive understanding of healthcare systems having worked with systems in Europe, North America, and Asia Pacific.

Anu Solanki, life sciences and biomaterials expert at PA Consulting, is a biomaterials scientist, with expertise in product development, tissue engineering, and regenerative medicine. She has taken several products from initial proof of concept to market evaluation, within the cell and gene therapy and medical device industries. Anu started her career developing acellular materials for tissue regeneration applications. Anu has a PhD in Biomaterials from Imperial College London, UK, developing regenerative medicine products for chronic wounds, and a Master’s in Materials Engineering from Queen Mary University, UK.

Dr Luca Pinton, life sciences and cell and gene therapy expert at PA Consulting, is a consultant with extensive expertise in R&D, biomedical research innovation, technology, and its translation into the clinical setting. Luca graduated in Pharmacy with honours at University of Ferrara, Italy, after undertaking a research internship at Karolinska Institute in Sweden. After, Luca completed a PhD in Stem Cell Biology & Regenerative Medicine at King's College London and UCL, UK. In his five years of research experience, Luca received more than 10 awards, authored seven publications, and presented at over 15 international conferences, in the areas of neuromuscular and CNS disorders. Luca was also Deputy Head and Business Development Director at Innovation Forum London and Senior Coordinator at the London Stem Cell Network.
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Hilary Thomas
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Anu Solanki
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Dr Luca Pinton
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