Gene-fixing treatments have now cured a number of patients with cancer and rare diseases.
Genes are composed of DNA that contains necessary information for making proteins that are vital for the human body to function optimally. Some gene mutations result in these proteins not being made correctly and can lead to genetic disorders. Gene therapy works by repairing, repressing, or replacing dysfunctional genes that cause disease with the aim of reestablishing normal function. Gene therapy is an attractive area for drug development because with the right target and approach, it can address the root cause of a severe disease. For certain disorders where known genetic mutations lead to deficient or non-functional protein production, gene therapy can fix the underlying defect and provide a path to produce the functional protein.
Gene therapy—the idea of modifying a person’s DNA to treat disease—represents a major shift in medicine. Instead of just treating symptoms like the vast majority of drugs on the market, gene therapy aims to correct the underlying genetic cause of a disease. Doctors and scientists hope these treatments will be a one-shot cure.
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Last year, we wrote that 2016 was gene therapy’s most promising year. But 2017 proved to be even bigger. Gene therapy had a hell of a 2017. After decades of promises but failed deliveries, last year saw the field hitting a series of astonishing home runs.
The concept of gene therapy is elegant: like computer bugs, faulty letters in the human genome can be edited and replaced with healthy ones.
But despite early enthusiasm, the field has suffered one setback after another. At the turn of the century, the death of an 18-year-old patient with inherited liver disease after an experimental gene therapy treatment put the entire field into a deep freeze.
But no more. Last year marked the birth of gene therapy 2.0, in which the experimental dream finally became a clinical reality. Here’s how the tech grew into its explosive potential—and a sneak peek at what’s on the horizon for 2018.
The global gene therapy market was valued at $584 million in 2016, and is estimated to reach $4,402 million by 2023, registering a CAGR of 33.3% from 2017 to 2023. Due to the high success rate during the preclinical and clinical trial, gene therapy is gaining popularity. There are many techniques used for gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of gene whose function is abnormal.
Tumor suppressor and suicide gene therapy are expected to be a lucrative segment for the market players as they are among the fastest growing segment in the gene therapy market. The therapy involves the introduction of new genes into the tumor cells that help destroy the tumor cells without affecting the neighboring healthy cells.
How Does Gene Therapy Work?
Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.
A quality that is embedded straightforwardly into a cell more often than not does not work. Rather, a bearer called a vector is hereditarily built to convey the quality. Certain infections are frequently utilized as vectors since they can convey the new quality by contaminating the cell. The infections are adjusted so they can’t cause sickness when utilized as a part of individuals. A few kinds of infection, for example, retroviruses, incorporate their hereditary material (counting the new quality) into a chromosome in the human cell. Different infections, for example, adenoviruses, bring their DNA into the core of the phone, however the DNA isn’t incorporated into a chromosome.
The vector can be infused or given intravenously (by IV) specifically into a particular tissue in the body, where it is taken up by singular cells. On the other hand, an example of the patient’s cells can be evacuated and presented to the vector in a research facility setting. The cells containing the vector are then come back to the patient. On the off chance that the treatment is fruitful, the new quality conveyed by the vector will make a working protein.
Specialists must defeat numerous specialized difficulties previously quality treatment will be a reasonable way to deal with treating ailment. For instance, researchers must discover better approaches to convey qualities and target them to specific cells. They should likewise guarantee that new qualities are absolutely controlled by the body.
The key companies profiled in the report include Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc., Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica, and NewLink Genetics Corp.
Other prominent players in the value chain include Amgen, Epeius Biotechnologies, Abeona Therapeutics, Sanofi, UniQure, Juno Therapeutics, Adaptimmune, Celgene, and Advantagene.
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