A mere look around and you will find people with different physical appearances and behaviours, like they vary in their complexion, color of their hair, height, body structure etc. They survive and behave differently in a specific situational setting. It is all due to their distinct gene structure.
These differences are either passed down from parents to their offspring or result later in the course of their lives due to exposure to radiation, drug intake, exposure to ultraviolet rays or viral infections. These genetic differences can explain why some people develop certain diseases and others do not.
Incorrect alterations in genes can give rise to the formation of proteins that are unable to perform their designated functions effectively. These genetic anomalies, known as gene mutations, have the potential to result in various genetic disorders. Illnesses such as sickle-cell Anaemia, Haemophilia, Huntington's disease and more are direct consequences of the gene mutations.
To treat or even prevent such genetic diseases at their source, gene therapy has emerged as a remarkable scientific innovation. Rather than relying on traditional methods such as medication or surgery, gene therapy techniques enable doctors to modify an individual's genetic composition, thereby offering a unique approach for treating genetic disorders.
Gene therapy is all about replacing a faulty or absent gene with a healthy and functional copy, or turning off a dysfunctional gene, or introducing a new gene into cells. The end motive is to effectively restore proper gene function.
Gene therapy relies on specialised carriers, such as viruses and bacteria, to accomplish its objectives. These vehicles are genetically modified to ensure they are non-infectious, thus serving as vectors, transporting essential genes to the precise locations where they are lacking; like Roctavian, for instance. Recently the US Food and Drug Administration (FDA) approved this gene therapeutic drug for patients afflicted with Haemophilia A, a condition characterised by inadequate blood clotting, resulting in uncontrollable and spontaneous bleeding. To treat this life threatening condition, Roctavian is administered intravenously, wherein neutralised viruses are dispatched to the patient's liver. Within the liver, these viruses seamlessly integrate the gene responsible for generating a clotting protein, thereby facilitating the restoration of normal blood clotting function.
Likewise, gene therapy is a promising treatment for other diseases including sickle cell disease, an eye disorder called Leber congenital amaurosis, a muscle disorder called spinal muscular atrophy, as well as acquired disorders like leukaemia.
With an aim to revolutionise the medical & healthcare sectors globally, the London Biotechnology Show will host a world-class exhibition and a comprehensive conference that will bring together leading policymakers, thought leaders, investors, industry experts, startups and other key stakeholders to network at the highest level, showcase innovative solutions and deliberate upon the current trends and future of gene therapeutics.