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Gene Therapy: The “Hunt” for CNS disorders!
Many companies have tried over the years to find a way to reverse the course of crippling central nervous system disorders like amyotrophic lateral sclerosis (a.k.a. Lou Gehrig’s disease) but have failed. Fortunately, Gene therapy has proven to be an answer to a wide range of serious central nervous system disorders like Lou Gehrig’s disease, Friedreich’s Ataxia, and Parkinson’s etc.
About Gene Therapy
Gene therapy involves the use of a modified virus—a viral vector—as a vehicle to deliver healthy genes into a cell to replace faulty or missing ones. This is a new method with potential for treating a broad range of acquired and inherited neurologic diseases, where the causative gene defect or deletion has been identified. In addition to gene replacement the application of gene products that reduce cellular dysfunction or death represent new therapeutic options. Inspite of the significant setbacks and limited success that the field of gene therapy has experienced, it has the potential to succeed where so many drugs have failed by providing a way to either cure, or significantly reverse the effects of these enervating diseases.
Gene Therapy: Role in CNS
The potential of Gene therapy in the field of Central Nervous System disorders has been propelled by genetic technologies, including identifying disease genes and disruptive mutations, design of genomic interacting elements to regulate transcription etc. These versatile new tools for manipulation of genetic elements provide the ability to tailor the mode of genetic intervention to specific aspects of a disease state.
The reasons for Central Nervous System based disorders to become a preferred area of application for gene therapy is the number of rare genetic disorders affecting the brain that don’t have any effective treatments. Moreover, a variety of gene delivery methods now provide strong scientific underpinnings for the design and implementation of powerful therapeutic strategies that can tackle focal as well as global disease. Additionally, nervous system offers several practical advantages for intervention, including a reduced immune response to vectors and transgenes in the brain parenchyma as compared with other peripheral sites of injection, the presence of only a few normal replicating cells that might be transformed by vector integration into their genome, and the reduced ability of vectors injected into the brain to access germline tissues as compared with peripheral injections. However, insufficient resources and funds are proving as a hurdle resulting in marginal success in this field. Inspite of this, there is much optimism and confidence in the potential of Gene therapy to develop an effective treatment for neurologic diseases 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 UniQure Biopharma, Sanofi, Oxford BioMedica etc., are operating in the field of Central Nervous System disorders in the gene therapy domain. A growing number of partnership between companies in drug development for example between Audentes and Genethon etc., are driving the new gene therapy research. The industry’s collective pipeline is brimming with 300+ therapies of which CNS collectively accounts for 12 % of gene therapies in pipeline. The global market opportunities for gene therapy have risen drastically and the tide has been turning back to enthusiastic support.
DelveInsight’s Gene Therapy Reports
DelveInsight’s Gene Therapy Reports cover the entire gene therapy market insights for central nervous system based 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.