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Advancements in gene therapy: A promising future for rare disease treatment
Thought leadership article by Georg Schroeckenfuchs, head — Gulf and Saudi Country Group, Innovative Medicines at Novartis
In the realm of medical breakthroughs, few areas hold as much promise and potential as gene therapy. The concept of manipulating genes to treat diseases was once confined to the realm of science fiction. However, recent advancements have turned this dream into a tangible reality, offering hope to millions of individuals afflicted by rare diseases.
Because rare diseases affect such a small percentage of the population, they can often go unnoticed or receive limited attention. Yet, for those affected, the impact can be life-altering. Recognising the urgent need for innovative solutions, scientists and medical experts have been increasingly directing their focus toward gene therapy as a potential game-changer.
Gene therapy aims to manage certain diseases through various gene manipulation strategies such as gene replacement, addition, silencing, reprogramming, and editing by modifying or replacing defective genes with healthy ones, and targeting the underlying cause of a disease rather than merely managing symptoms. This ground-breaking approach offers the possibility of long-term, if not permanent, relief for patients with rare diseases. By addressing the root cause, gene therapy holds the potential to provide transformative effects and offer hope where previously there was none.
In the GCC region, significant advancements are being made in the realm of gene therapy. In 2020, we saw spinal muscular atrophy (SMA) gene therapy introduced for the first time into the UAE market. SMA is a genetic disease caused by a deletion or mutation in the Survival Motor Neuron 1 (SMN1) gene. It is characterised by progressive degeneration and irreversible loss of spinal motor neurons. Untreated, SMA is the leading genetic cause of infant mortality.
Beyond SMA, ongoing clinical trials and research initiatives are exploring the vast potential of gene therapy for various rare diseases, promising a revolutionary shift in the treatment landscape. As a global leader in gene therapy, Novartis has been spearheading this movement by leveraging its unparalleled expertise and dedication to scientific innovation.
With a deep commitment to research and development, Novartis is propelling breakthrough discoveries that have the potential to revolutionise the lives of patients. Last year, we signed an initial agreement with Saudi Arabia’s Ministry of Investment to expand local investment activities in multiple areas including cell and gene therapy. Novartis Saudi also signed an outcome-based risk-sharing MoU agreement with the Ministry of Health to improve patient access to innovative medicines by adding next-generation Novartis treatments into the National SMA Programme.
In the UAE, the Ministry of Health and Prevention’s introduction of innovation gene-based remedies has opened up new possibilities for the treatment of blindness caused by inherited retinal disease. In fact, it led to the extraordinary story of two sisters aged 13 and 11 becoming the first of several patients to receive vision-saving treatment. Delivering normal copies of the RPE65 gene to the eye, the groundbreaking genetic therapy allows for the production of vital proteins that help to preserve and even repair the tissue of the retina.
As advancements in gene therapy continue to unfold, the horizon for rare disease treatment appears increasingly promising. It’s a captivating and transformative movement that is bringing with it newfound hope for individuals living with rare diseases.
Certainly, if we can leverage the full potential of gene therapy, we can create a brighter future for patients in need.
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