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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 cure 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 personalized 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 technology used to deliver repaired genes into patients’ cells (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.

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

Our involvement

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

Our primary research program with TIGET is focused on a gene therapy to treat ADA-SCID (adenosine deaminase severe combined immunodeficiency) – a rare and devastating disease in which children have a severely deficient immune system because of a mutation in one gene. We are also investigating gene therapy for two other rare diseases: metachromatic leukodystrophy and Wiskott-Aldrich syndrome.

We have worked alongside the team at TIGET to advance their research, supporting the project with our expertise in medicines development, manufacturing, and the global regulatory environment to turn an academic-led procedure into what could become an approved therapy for patients globally.

We are also expanding our gene therapy activities beyond rare diseases. In 2014, we entered into 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 specialized medical procedures to treat individuals - is increasing for a wide range of disorders.

Our vision for cell and gene therapy

At GSK we are building our cell and gene therapy capabilities to help more patients by investing in a dedicated, multi-disciplinary group within our R&D organization.

This area of science requires specialized skills and experience to rapidly progress the novel technology, establish new production techniques, engage regulatory authorities on new 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 make this technology a reality for patients.

As opportunities in this field continue to emerge in areas beyond rare diseases 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 new 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.