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News from News-Medical.net Apr.
26, 2007
New biotech company develops
UD-patented technologies for repairing genes
Devices/Technology Published: Thursday, 26-Apr-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.
http://www.udel.edu/
http://www.news-medical.net/?id=24180
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