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Cell and gene therapy: a new era of medicine

Since the structure of DNA was discovered by James Watson and Francis Crick in 1953, significant advances have been made in our understanding of diseases caused by faulty genes. 

This has led scientists to ask, “If we can pinpoint a faulty gene and correct it, could we potentially find a way to treat the genetic diseases which are so devastating for patients and their families?”

Answering this question is something scientists have been attempting to do in the rapidly developing field of cell and gene therapy - a groundbreaking approach to personalised medicine that involves repairing or replacing defective genes, cells or tissue using cutting edge techniques.

Pioneering research

In development for decades, the research hasn’t been without its scientific setbacks. In the 1990s, when research was in its infancy, some patients who received investigational gene therapies died as result of the therapy.

Tragically, the first patient died following an immune reaction to a virus that had been prepared to deliver the therapeutic gene. Others developed leukaemia when a version of a virus used to "inject" cells with the correct DNA inadvertently "turned on” cancer-causing genes.

This was a major setback. However, the perseverance of scientists around the world is increasing confidence that cell and gene therapy could be coming of age.

Encouraging results have been seen in certain cancer immunotherapies and diseases including haemophilia B, lysosomal storage disorders and various other rare diseases.

Our involvement

We have been involved in cell and gene therapy since 2010, working with Fondazione Telethon and the Ospedale San Raffaele, acting through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), a world-leading research centre for stem cell gene therapy in Italy. SR-TIGET has been a pioneer in bringing gene therapy from preclinical studies to research in patients.

Our primary research programme with SR-TIGET is focused on ADA-SCID (adenosine deaminase severe combined immunodeficiency) – a ultra rare and devastating disease in which children do not have a healthy, fully-functioning immune system and so are unable to fight off every day infections.

A child develops ADA-SCID by inheriting two faulty copies of the ADA gene, one copy from each parent. We are also investigating two other rare diseases - Metachromatic Leukodystrophy and Wiskott-Aldrich syndrome.

We are working alongside the team at SR-TIGET to continue their research, supporting the project with our expertise in medicines development, manufacturing and the global regulatory environment.

We are also expanding our development activities beyond rare diseases. In 2014, we entered into a collaboration with Adaptimmune, a specialist research company exploring the potential of gene therapy to improve the ability of the immune system to fight cancer.

Cell and gene therapy is a world apart from the ‘small white pill’ made in a factory and prescribed to patients through their pharmacy. Yet the potential of applying this approach – using a patient’s own cells and highly specialised medical procedures to treat individuals - is increasing for a wide range of disorders.

Our vision for cell and gene therapy

We are building our cell and gene therapy capabilities with the aim of helping more patients by investing in a dedicated, multi-disciplinary group within our R&D organisation.

This area of science requires specialised skills and experience to rapidly progress the novel technology, establish new production techniques, engage regulatory authorities on development and registration strategies, address logistical challenges in the supply chain and identify ways to cost-effectively scale-up a very complex scientific process.

All these elements are necessary to potentially make this technology a reality for patients.

As opportunities in this field continue to emerge in areas beyond rare disease and cancer, our vision is for cell and gene therapy to stand alongside the more commonly used small molecule and biopharmaceutical approaches in the development of medicines.

This demands a rapid shift in thinking, but we believe that the potential of this technology to help patients is now clear. A new chapter in the story that was started by Watson and Crick has begun.