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Gene Therapy: The “Hope” for Immunologic disorders!
About Immunologic disorders
Immunological disorders are a dysfunction of the immune system. These disorders cause abnormally low activity or overactivity of the immune system. In cases of immune system overactivity, the body attacks and damages its own tissues. These diseases decrease the body’s ability to fight invaders, causing vulnerability to infections. Most immunological diseases are caused by errors (mutations) in specific genes. It has long been the hope that one day it would be possible to cure these diseases by fixing the mutation that causes the disease and thus affect a cure. As a result of the human genome project and similar efforts to map all of the genes present in human beings, we now know the identities of the specific genes involved in many diseases, including the vast majority of immunological diseases. We have finally reached the stage where that long held hope is becoming a reality.
Gene Therapy: Role in Immunological disorders
In its broadest sense, gene therapy is the transfer of a gene, or genes to patients, for therapeutic purposes by using vectors as agents of gene transfer and gene expression. Traditional pharmacological approaches to treatment entail the synthesis of small, diffusable compounds given orally or by injection. These approaches have yet to provide ideal agents for use in immunological diseases. Recent research, however, has identified a number of proteins with the potential to improve treatment, but these are difficult to administer long term. Gene therapy provides the opportunity to deliver protein products, as well as therapeutic species of nucleic acids, such as antisense RNA, much more efficiently than traditional methods of drug therapy. Gene therapy’s greatest strength is its ability to produce high, sustained concentrations of therapeutic macromolecules within a defined anatomical location. Thus, making gene therapy an ideal candidate for treating immunological disorders.
Gene Therapy: The Market Scenario
Collectively, immunological disorders constitute a major, unmet, clinical challenge. Although no single disorder is highly prevalent, there are over 150 of them. Because these diseases are generally incurable and difficult to manage, there is a pressing need for novel approaches to their treatment. As a result the market opportunities for gene therapy in the field of immunological disorders have been reinvigorated by the entry of large biopharmaceutical companies such as Glaxosmithkline, Novartis. etc. which are operating in this domain. Many companies have started investing in and researching on gene therapy as it has the potential to transform many areas of immunological disorders, leading to more rapid development timelines and access to better treatment options within a shorter time frame. However, there are some risks that need to be overcome and safer vectors need to be developed. Various laboratories around the world are working at modifications of the viral vectors in order to improve their safety. Nevertheless, gene therapy must still be regarded as an experimental therapy. It is likely that the inherent problems will be worked out in the coming years and that a larger number of immunological diseases will be cured by gene therapy.
DelveInsight’s Gene Therapy Reports
DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for immunological disorders including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners in this therapeutic area. It also identifies the potential market movers and future regulatory landscape.