Is gene therapy the beginning of a revolution in medicine? (Guest blog)

Ever since the Human Genome Project started unravelling the mysteries of our genes in the 1990s, a lot of hope has been pinned on the potential of gene therapy as a new era of medicine. The idea is that by fixing, replacing or avoiding the expression of a faulty gene – genetic disorders can be treated or even cured.

Personally, I believe that gene therapy could revolutionize medicine and treatment prospects for thousands of people living with rare diseases, especially when you consider that 80% of rare diseases have a genetic component.

There have been incredible advances in the last few years in terms of vector engineering, disease modelling and both clinical and commercial-scale manufacturing – all of which are essential to ensure that the full potential of this approach is realized for as many patients as possible.

So, what are we doing to progress these therapies from lab to patients? Pfizer has chosen to focus on an in vivo adeno-associated-virus (AAV)-mediated gene addition or gene transfer delivery method.

The process of AAV includes targeting the missing or non-functioning genes in an individual’s DNA, adding to it or replacing it with normal functioning genes. The technology involves the introduction of genetic material (DNA or RNA) into the body, often by delivering a corrected copy of a gene to a patient’s cells to compensate for a defective one, using a viral vector. This technique could potentially address a variety of genetic diseases.

The ambition of gene therapy is to restore normal function of cells, potentially enabling a patient to manage his or her disease without the need for ongoing treatments. For example, hemophilia patients require multiple injections each month to manage their condition. My dream is to reach a stage where children and their parents can focus on living life to the fullest without the burden of ongoing treatment or the worry of a bleeding episode.

I believe one of the most exciting things about gene therapy is the potential to restore a true sense of freedom to those living with conditions such as hemophilia. It’s not just about being able to increase physical activity or sports and travel opportunities but simply to be able to do the everyday things that most of us take for granted, free from pain and anxiety – can you imagine what that could mean? However, it is still early days and while gene therapy holds promise for people with genetic diseases, it may not be an appropriate solution for everyone. We will have to wait and see.

That said, gene therapy is undoubtedly one of the most important and exciting areas to work in and, despite the uncertainties, I think this is the beginning of a new era of medicine. I’m ready to be a part of it, are you?

Reda Guiha

Regional President, International Developed Markets (IDM), Pfizer Rare Disease
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