What Is Fanconi Anemia?
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?
The Opportunity for DGI
The availability of the University of Minnesotas 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
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.
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