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The Fanconi Anemia Clinical Trial —

We are in the process of filing an Initial New Drug (IND) application as we prepare for clinical trials for Fanconi Anemia, or FA, a rare, fatal orphan disease. Our success with FA opens the door for the use of DGI’s proprietary, safe, long-lasting gene therapy in a wide variety of diseases, including Hemophilia, cancer, ischemia and AIDS.


What Is Fanconi Anemia?
Fanconi Anemia (FA) is one of the inherited anemias that leads to bone marrow failure (aplastic anemia). Though considered primarily a blood disease, it may affect all systems of the body. Many patients do not reach adulthood.

There are at least seven genes responsible for FA. Preclinical work on two of the FA therapeutic genes (FancA and FancC) accounting for 80% of the incidence of FA in the US, has been conducted by DGI under a NIH grant.

How Is Fanconi Anemia Treated?
At the present time the only long-term cure for the blood defects in FA is a bone marrow transplant from an exact-match sibling. This treatment has many risks associated with it, and the risks are compounded in FA patients because of their extreme sensitivity to radiation and chemotherapy.

The Opportunity for DGI —
The most experienced physicians in the world for the treatment of Fanconi Anemia are professors at the University of Minnesota Medical School. We were approached by these physicians, who had found that our gene therapy technology was ready for clinical testing in FancC deficient Fanconi Anemia patients.

The availability of the University of Minnesota’s Molecular and Cellular Therapeutics facility for the preparation of clinical-grade material, along with the trial being directed by the most experienced physicians in the world for the treatment of FA, is a unique opportunity.

This orphan disease trial can be done with a low number of patients. This permits us to complete the planned work quickly and at a very low cost. While the market for FA gene therapy is small, we can quickly achieve a significant market share generating significant revenues.

DGI's FA Clinical Trial —
The clinical trials will be conducted with small but meaningful sample sizes — 10 patients for Phase I and Phase II.

DGI's FA Clinical Gene Therapy Process

The rare orphan nature of this disease (with a prevalence of only 3,000 patients/year) and our collaboration with the University of Minnesota (an international center for the treatment of FA) means that we can get FDA approval, conduct clinical trials and begin commercial sales within four years. Tax breaks are available for investors.

For more information about Fanconi Anemia, see the Fanconi Anemia Research Fund web site.

The Fanconi Anemia Research Fund


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