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About Gene Therapy

Gene Therapy: Turning Point

Gene therapy is an approach to treat a disease by introducing specific genes into a patient’s body to obtain the physiological functions of proteins generated by the gene expression.

The first gene therapy was conducted in the U.S. in 1990 for patients with ADA deficiency, a congenital immunodeficiency. Since then, more than 2,000 gene therapy clinical studies for diseases including cancer and HIV have been performed around the world.. In Japan, more than 30 gene therapy clinical studies have been conducted since the first gene therapy treatment was done for a patient with ADA deficiency at Hokkaido University in 1995.

Genes exist in every cell of the human body, and control how well our body functions by turning on or off the genetic switches. When a genetic switch is turned on, the sequencing of DNA is copied to a messenger/transcription molecule known as mRNA. Based on the genetic information transcribed in mRNA, enzyme, hormones and other proteins are produced. These proteins yield various physiological effects to support the healthy condition of the body. Today, many of the conventional drugs are low-molecular compounds which obtain therapeutic efficacy by affecting the way in which proteins react. Gene therapy, on the other hand, has a completely different mechanism of action to that of a low-molecular compound, and works on the highest level of a chain of gene and protein expression.

Today, more than 20 years after the first clinical trials, gene therapy is facing a huge turning point. First, the target disease area for gene therapy has expanded. Second, there has been an improvement in the vector technology used in delivering genes to an affected area. 

Mechanisms of Genes and Target of Drugs

Mechanism of genes and target of drugs

Expanding the Target Group of Patients

Gene therapy was first introduced to treat very serious genetic disorders. It demonstrates therapeutic effectiveness by introducing a specific gene expression to a patient who has a specific gene defect. Gene therapy is also expected to be a treatment for cancers with no effective treatments established, and it has been developed with such methods as cancer destruction, and enhancement of cancer specific immunity. Currently, there are some technical hurdles to overcome, such as the difficulty in introduction of gene expression only in specific cancer cells. However, there are many oncology patients and therefore, a number of biotech companies are committed to the research and development of gene therapy drugs for various cancers.

Gene therapy in treating lifestyle-related conditions is also receiving attention. AnGes is developing genetic medicine for lifestyle-related conditions such as vascular diseases in the lower limbs and the heart. Due to the rapidly aging population and changing eating habits, the number of patients suffering from lifestyle-related diseases is increasing worldwide, making the development of highly effective medicine to cope with these disorders a matter of urgency. The high demand in this area offers considerable business opportunities.

Gene Therapy and Vector Technology

Even though gene therapy promises breakthrough results, there is a concern about adverse effects. For example, it was reported in autumn 2002 that a gene therapy case in France produced leukemia. In the last several years, however, genetic medicine has greatly expanded in the methods of introducing a genetic expression, as well as other parameters regarding the target patient group, and now it is no longer feasible to evaluate the general situation only on the basis of individual examples of genetic therapy.  The second stream here is a transition of vector technology, which is largely related to concerns regarding the adverse effects.

Referring to the case in France above, a genetic expression was introduced to the bone marrow tissue of a patient (suffering from a congenital genetic disease) with a retrovirus vector. A retrovirus randomly recombines DNA (chromosomes). This is presumed to be the cause of the adverse effects.

AnGes’s HGF Plasmid is different from the above explained gene therapies with virus vectors since it does not use virus itself as the vector. This prevents the possibility of a gene being introduced to a DNA sequence, also eliminates other virus-related adverse effects such as infection. On the other hand, many achievements have been made in virus vector safety in recent years, and a gene therapy for lipoprotein lipase deficiency using adeno-associated virus vector has been approved in Europe as a first gene therapy product in developed countries.


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