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When a genetic medication introduces a genetic expression into an afflicted patient's body, the disease is cured due to the physiological functions of proteins generated by such a genetic expression. The first genetic medicine was developed for ADA deficiency - congenital immunodeficiency - patients in the U.S. in 1990. Since then, genetic medication has been used in approximately 4000 cases around the world, including cancer, HIV, etc. In 1995, Hokkaido University implemented genetic therapy to heal a patient with ADA deficiency for the first time in Japan, anti-cancer therapies of the Institute of Medical Science Society at Tokyo University as well as at Okayama University followed.

The switches determining how well our functions work are turned on and off inside our cells throughout the body. When such a switch is turned on, the genetic sequencing of DNA is copied to a messenger/transcription molecule known as m-RNA. The next step is preparing amino-acids, hormones, and other (poly) proteins on the basis of information transcribed in m-RNA (a process of translation). These proteins yield various physiological effects to support the healthy condition of the body. At present, low-molecular compounds affecting the way in which proteins react are the medications primarily used. Since genetic medication, on the contrary, works on the highest level of a chain by obtaining proteins as per genetic expression, its mechanism is completely different to that of a low-molecular compound.

Today, 10 years after the first trials, genetic medication is proving to be a big turning point in medicine. The target group of patients is expanding, on the other vecter techonogy which introduceds a genetic expassion to an affected area, has to be further improved. Our company takes the lead in dealing with both issues.


Mechanism of genetic and target agents
Mechanism of genetic and target agents


Expanding the target group of patients
Genetic medication was first introduced to heal a very serious genetic disorder. Due to introduction of a specific gene expression to an afflicted patient's body, a successful therapeutic effect can be expected. Cancer, with no effective remedy, is a field where the development of new medical treatments applying genetic medication are highly anticipated. Methods of cancer cell destruction, as well as methods of immunity enhancement against cancer cells, are being studied. Currently, there are some technical hurdles to overcome, such as the difficulty in the targeted introduction of genes expressions only against specific cancer cells. However, this field involves many patients, and many venture companies are committed to the research and development of genetic medication for various cancer types.

In addition to these projects, our company is developing genetic medication for lifestyle-related and chronic conditions - the focus of much attention recently. Such therapy is intended for lesions of blood vessels in the lower limbs or the heart and chronic articular rheumatism. Due to such phenomena as the rapidly aging population and changing food habits, the number of patients suffering from lifestyle-related and chronic conditions is increasing, making the development of highly effective medicine to cope with these disorders a matter of urgency. Demand being huge, this area offers considerable business opportunities.


Genetic medicine and vector technology
Even though genetic medication promises breakthrough results, there is a concern about adverse effects. For example, using genetic medication in France in Autumn 2002 produced leukemia. In the last several years, however, methods of introducing a genetic expression, as well as other parameters regarding the target patient group, have expanded greatly, 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 recombines DNA (chromosomes) at random. This is presumed to be the cause of the adverse effects.

Generally, there are various doubts regarding the safety of genetic medications. Some of these are: Are there any negative effects (related to a reproduction cell) on offspring? Will genes be manipulated?

All of these originate in the introduction of a gene to a chromosome when a retrovirus vector is used. Using a virus vector poses even more hazards: contamination and/or immunological reaction. It is only in cases of a very critical congenital disease that presently we are forced to use methods posing such risks.


HGF genetic medication with virus vectors
Our company's genetic medication for ischemic disorders is different from all conventional therapies in that it does not use any vectors at all. This
prevents the possibility of a gene being introduced to a DNA sequence,
also eliminating all other adverse effects such as virus gene-to-chromosome integration. We use a method of supplying the gene expression (known as the HGF, a gene formed in our bodies) from the outside.

We believe the safety guarantee to be the most important factor in the proliferation of genetic medication. With this in mind, we are developing non-viral HVJ envelope vectors - highly safe technology with a completely extracted viral genome. Multiple venture enterprises, including our company, are now developing gene-introduction technologies which do not use virus vectors.

Applications of genetic medicaton range from treatment of specific diseases using virus vectors to safely treating general conditions without using virus vectors. AnGes MG is always on the forefront of developments.


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