OrphageniX Inc., a new biotechnology company founded by University of Delaware researchers, has been established in Wilmington to develop and commercialize UD-patented technologies for repairing genes that cause rare, hereditary diseases such as sickle cell anemia and spinal muscular atrophy.
The announcement was made in a news release issued by the company on April 13.
Eric Kmiec, professor of biological sciences, and Hetal Parekh-Olmedo, senior research associate, both in the UD College of Arts and Sciences, co-founded and incubated OrphageniX at UD's Delaware Biotechnology Institute in the Delaware Technology Park in 2005.
Kmiec holds 14 UD patents for gene-editing technologies and is widely regarded as a pioneer in the field.
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).
Kmiec and Parekh-Olmedo discovered a way to introduce a tiny fragment of DNA into a diseased cell to replace the defective portion, triggering the cell to heal itself.
This method, which focuses on correcting a patient's genes to make their own proteins, offers a safer approach than treating a patient's genes with foreign genes or protein replacements, and eventually may lead to cures for rare diseases, according to Michael Herr, president and chief executive officer of OrphageniX.
Herr previously was the director of science and technology at the University City Science Center of Philadelphia.
Sickle cell anemia and spinal muscular atrophy are among the diseases that OrphageniX is targeting, according to Herr.