Gene Therapy to “reverse” Metabolic disorders!

Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the risks of experimental therapy are outweighed by potential benefits to trial participants.

About Gene Therapy

Gene therapy is defined as the treatment or cure of human diseases by transfer of nucleic acids (DNA or RNA) to affected cells and it covers a broad range of therapeutic applications from Mendelian disorders to complex metabolic diseases. Most diseases caused by genetic deficiencies could in theory, be treated by the introduction and expression of a normal gene into an appropriate target tissue. It seems like that gene therapy strategies for most metabolic disorders will not require strict gene regulation, as a fraction of the normal levels of gene activity could result in amelioration or significant improvement in the clinical outcome.

Metabolic disorders refer to different types of medical conditions caused by genetic defects — most commonly inherited from both parents — that interfere with the body’s metabolism. Metabolism is the complex set of chemical reactions that your body uses to maintain life, including energy production. When these chemical processes don’t work properly due to a hormone or enzyme deficiency, a metabolic disorder occurs. Inherited metabolic disorders fall into different categories, depending on the specific substance and whether it builds up in harmful amounts (because it can’t be broken down), it’s too low, or it’s missing. Metabolic disorders affects millions of people world-wide and is the fourth or fifth leading cause of death in the developed world. Gene therapy is an experimental form of treatment that aims to use the transcriptional machinery of the patient to produce the active factor that exerts the intended therapeutic effect, ideally in a permanent, tissue-specific and manageable way.

Gene Therapy: Role in Metabolic disorders

Genetic manipulations for altering gene expression pattern in vivo with therapeutic outcome, known as gene therapy, offers a fundamentally different approach to pharmacotherapy for inherited as well as acquired metabolic disorders. Current and possible candidates for gene therapy in the field of metabolic disorders include Sanfilippo type A syndrome, Metachromatic Leukodystrophy (MLD), Crigler-Najjar syndrome, Urea cycle disorders and Diabetic Macular Edema etc. because of their poor prognosis.

Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transfer technologies. However, interspecies differences in disease biology, vector performance, host–vector interactions and increased size with the associated necessity to functionally correct a greater number of cells to reach the threshold required for therapeutic benefit are acting as biological limitations in this field. Despite these challenges, it is predictable that the gene therapy based strategy in modulating metabolism and treating metabolic disorders will surely impact how we live a healthy life in the near future.

Gene Therapy: The Market Scenario

Many companies are investing in and researching on this field using gene therapy due to its promising effects. Large Pharmaceutical and Biotech giants, such as GlaxoSmithKline, Pfizer, and Alnylam etc., are operating in the field of metabolic disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Lysogene and RegenX Biosciences etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies for various therapeutic areas. Gene therapy has the potential of providing a definitive cure for patients with these diseases. The global market opportunities for gene therapy have risen drastically indicating that there’s big money in gene therapy.

DelveInsight’s Gene Therapy Reports

DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for metabolic 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.