New biotech company to commercialize novel UD gene-repair technology

April 24, 2007

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.

Sickle cell anemia affects an estimated 72,000 Americans, mostly African Americans. Those afflicted with the disease produce sickle- or crescent-shaped blood cells instead of smooth, round blood cells. These "sickle cells" tend to get stuck in the blood vessels, blocking the flow of blood to the limbs and organs, often causing pain, organ damage and anemia in the process.

Spinal muscular atrophy is a genetic disease caused by the progressive degeneration of motor neurons in the spinal cord, resulting in weakness and wasting of the voluntary muscles. Weakness is often more severe in the legs than in the arms. The disease affects approximately one in 6,000 babies, and about one in 40 people are genetic carriers, according to Families of Spinal Muscular Atrophy.

Herr said the company's immediate strategy is to advance the new technologies to clinical trials, assemble a leadership team around its UD founders, and identify strategic partners.
OrphageniX is located at 300 Water Street in Wilmington. The company's web site is

University of Delaware

Related DNA Articles from Brightsurf:

A new twist on DNA origami
A team* of scientists from ASU and Shanghai Jiao Tong University (SJTU) led by Hao Yan, ASU's Milton Glick Professor in the School of Molecular Sciences, and director of the ASU Biodesign Institute's Center for Molecular Design and Biomimetics, has just announced the creation of a new type of meta-DNA structures that will open up the fields of optoelectronics (including information storage and encryption) as well as synthetic biology.

Solving a DNA mystery
''A watched pot never boils,'' as the saying goes, but that was not the case for UC Santa Barbara researchers watching a ''pot'' of liquids formed from DNA.

Junk DNA might be really, really useful for biocomputing
When you don't understand how things work, it's not unusual to think of them as just plain old junk.

Designing DNA from scratch: Engineering the functions of micrometer-sized DNA droplets
Scientists at Tokyo Institute of Technology (Tokyo Tech) have constructed ''DNA droplets'' comprising designed DNA nanostructures.

Does DNA in the water tell us how many fish are there?
Researchers have developed a new non-invasive method to count individual fish by measuring the concentration of environmental DNA in the water, which could be applied for quantitative monitoring of aquatic ecosystems.

Zigzag DNA
How the cell organizes DNA into tightly packed chromosomes. Nature publication by Delft University of Technology and EMBL Heidelberg.

Scientists now know what DNA's chaperone looks like
Researchers have discovered the structure of the FACT protein -- a mysterious protein central to the functioning of DNA.

DNA is like everything else: it's not what you have, but how you use it
A new paradigm for reading out genetic information in DNA is described by Dr.

A new spin on DNA
For decades, researchers have chased ways to study biological machines.

From face to DNA: New method aims to improve match between DNA sample and face database
Predicting what someone's face looks like based on a DNA sample remains a hard nut to crack for science.

Read More: DNA News and DNA Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to