OrphageniX has been spun out from the University of Delaware by Professor Eric Kmiec and Hetal Parekh-Olmedo. Kniec holds 14 patents for gene-editing technologies and is widely regarded as a pioneer in the field. "Gene repair is focused on correcting a patient's own genes to make their own proteins, which should make gene repair a safer approach than treating with foreign genes or protein replacements," said Michael Herr, the new CEO of OrphageniX. "The innovative gene repair technology holds promise to eventually cure, not just to treat, these significant diseases." There are more than 5,000 rare or 'orphan' diseases, so named because each affects fewer than 200,000 people nationwide. A number of these diseases are caused by a single-point mutation in a gene--which is like a spelling error, a single "letter" out of place, in its DNA code. The DNA nucleotide adenine (A), for example, might be replaced by guanine (G), cytosine (C) or thymidine (T). The two co-founders of the company discovered a way to introduce a small fragment of DNA into a diseased cell to replace the defective portion. This can enable the cell to being healing itself. "Our immediate strategy is to advance to clinical trials, assemble a leadership team around our renowned Scientific Founders, and identify strategic partners." said Herr.
