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James Miskin Ph.D

Chief Technical Officer, Oxford Biomedica

New treatments and approaches for tackling serious diseases are being developed using cell and gene therapy.

Cell and gene therapy is adding a new dimension to the way serious diseases are being tackled in modern healthcare.

But these advanced treatments require complex research, development and manufacturing processes.

While the two are related, and to a degree interchangeable, gene therapy typically uses a genetic delivery system, such as a viral based vector, to treat a particular disease by replacing a malfunctioning gene or introducing a new gene to alleviate the disease.

Cell therapy, meanwhile, works in a similar manner but is often administered by modifying cells outside the body, with these modified cells being returned to the body to treat the disease.

Developing partnerships

James Miskin, who has more than 20 years’ experience in the field, says that one of the biggest challenges lies in ensuring the technology and viral based vectors – tools commonly used to deliver genetic material into cells – are safe.

Once that has been tackled, the next challenge lies in making enough of the vector to modify enough cells to treat the disease, explains Dr Miskin, Chief Technical Officer of Oxford Biomedica, a gene and cell therapy company specialising in the development of gene-based medicines.

As the therapies are complex, Dr Miskin points to the value of developing partnerships with other organisations, joining forces to help increase the chance of a product getting to market.

“Our partners benefit from our expertise on vector and cell engineering, analytical development, process development and regulatory interactions with different agencies globally,” says Dr Miskin, who joined Oxford Biomedica in 2000. He has overall responsibility for the company’s quality systems, analytical testing, lentiviral-based bioprocessing development and client programmes.

“Our own internal expertise is unique in that respect because we are also a product development company and were the first to administer a lentiviral vector based product, which many of the therapies we are developing utilise, directly into patients.”

Cell and gene therapy is adding a new dimension to the way serious diseases are being tackled in modern healthcare.

Tackling disease

Many of the therapies that are developed in the cell and gene therapy (CGT) field are in the form of a chimeric antigen receptor (CAR)-T-cells. This is where T cells (one of the main immune cells in the body) are modified to recognise cancerous cells and then to attack and kill them, thereby removing the cancer.

Oxford Biomedica makes the lentiviral vector that allows that genetic modification to occur to turn T-cells into cancer killing cells. It is also developing its own CAR-T cell programme targeting a particular tumour associated antigen and working with partners on therapies that target immune system diseases.

“We are using the same type of technology for gene therapy, by delivering the vector directly to patients as opposed to a cell therapy,” he says. “A good example of that is a therapy we initially developed for Parkinson’s disease, now being developed by a partner company Sio Gene Therapies, which is directly administered to the brain.”

COVID-19 vaccine

Oxford Biomedica also had a pivotal role in the manufacture of the AZ/Oxford COVID-19 vaccine, having joined the manufacturing consortium led by the Jenner Institute at the University of Oxford.

As the UK’s biggest company specialising in viral vector development, manufacture and analytics, they had already invested in innovation and manufacturing facilities and committed to the process at an early stage.

“We just accelerated our plans,” he says. “We went from never manufacturing an adenoviral vector at scale to now being the UK’s largest manufacturer of them.”

At the end of 2019, the company had three manufacturing suites. That has now increased to seven and it employs over 670 people, and many tens of millions of doses of the vaccine have been manufactured for AstraZeneca. “What COVID really highlights is the importance of companies with credible capabilities,” he says. “It is not just about clean rooms; you need the people, the knowledge and the expertise

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