300 Water Street, Suite 204
Wilmington, DE 19801

Phone: 267-334-5153 (mobile/office)
e-mail: michaelherr@verizon.net

OrphageniX is a drug development company with the mission to develop innovative therapeutics for genetic diseases with high unmet need using advancements in "Gene Editing" technologies. OrpageniX will focus on the low volume, high margin specialty pharmaceutical niche area of "Orphan Diseases" which are protected under the Orphan Drug Act (ODA). OrphageniX will benefit from ODA's multiple economic incentives that decrease drug development costs, expedite regulatory approval and guarantee prolonged market exclusivity for eligible disease indications. In addition, the OrphageniX technology can be used for diagnostics, research tools, plant and animal use and will be leveraged into these other areas by licensing the technology out, immediately creating additional value.

Gene Editing is a technology pioneered by Dr. Eric Kmiec, Founder of the company and Professor at the University of Delaware ( Delaware Biotechnology Institute). Gene Editing uses the cell's innate repair mechanisms to repair mutations that result in genetic diseases. It is similar to gene therapy, but avoids the risks associated with a viral vector. It works like anti-sense and RNAi, but is more efficient and permanent within the repaired cell because it changes the gene itself and not the transient message. Moreover, over the past decade, the technology has been perfected, refined and validated. It is now undergoing final validation in the hands of experts in genetic diseases in various academic institutions. With the formation of OrphageniX, the validation will reach the final phase prior to entering clinical trials. Orphagenix is a virtual company with major collaborations with Yale Medical School, Albert Einstein Medical Center and the University of Washington. OrphageniX has a very strong IP position in the field of genetic repair of mutations and is interested in partnering various disease indications with biotechnology and pharmaceutical companies..