Research&Development Therapeutics

Therapeutic approach





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In vivo genome editing therapy

ToolGen has been devoting all it’s competency to develop therapeutic CRISPR/Cas9 system to cure intractable genetic diseases which has no traditional medical meet.

ToolGen has accumulated competency to do gene correction, gene knock-in, gene knock-out with high credibility in terms of activity, specificity and low off-target.

ToolGen has been eager to set up it as CRISPR/Cas9 platform technology which can be applicable to variable diseases according to type of gene disorder. ToolGen has also set up AAV platform which enables delivery of CRISPR/Cas9 to eye, liver, nerve system with high specificity.

The extraordinary benefit of in-vivo CRISPR/Cas9 therapeutic platform is that just a time therapy can cure the diseases almost permanently.

Hemophilia is a man’s disease classified into Hemophilia A and Hemophilia B. Hemophilia A is caused by deficiency of clotting factor VIII and Hemophilia B is caused by deficiency of clotting factor IX. The incidence rates of two type are 1/12,000 and 1/30,000 respectively. Both type has no medical meet, so patient is depending on injecting clotting factors. This costs a patient around 160,000 (US$) per year, the sum of expenses worldwidely amounts to 10.8 billion(US$) for Hemophilia A and 6.7 billion(US$) for Hemophilia B.

Charcot–Marie–Tooth disease (CMT)
This heredietary genetic disease occurs in incidence of 1/25,000 and approximately 2.5 million people are under this disease worldwidely nowadays. This is one of the hereditary motor and sensory neuropathies with symptoms of progressive loss of muscle tissue and touch sensation across various parts of the body. Currently incurable, this disease need new therapeutic approach. The most frequent type is CMT1 which has incidence rate of 40% among entire patients and is caused by single mutation of specific gene(PMP22).

Ex vivo genome editing therapy

ToolGen has been developing Best-in-Class Cell therapeutics using CRISPR/Cas9 genome editing technology. Our therapeutic approaches which derived from combining immune cells and stem cells with CRISPR/Cas9 system show therapeutic effects beyond expecting. Even though it has long way to go, obstacles to overcome, this approach is shaping a new therapeutic paradigm and can be applicable to disease like cancer. ToolGen’s technology which focuses on way of elevating specific functions of cell in ex-vivo environment has expanding it's value as platform technology.

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