NaPro's Gene Editing Demonstrates Gene Alteration in Human Cells - Technology Promising for Sickle Cell Disease -
WASHINGTON, Jun 5, 2003 /PRNewswire-FirstCall via COMTEX/ -- NaPro
BioTherapeutics, Inc. (Nasdaq: NPRO) announces the development of a
clinically-applicable protocol to achieve specific gene alteration in human
hematopoietic stem/progenitor cells. These results, presented today at the
American Society of Gene Therapy (ASGT) Annual Meeting, achieve a milestone in
the development of its procedures for potential treatment of sickle cell
disease. Specifically, these data demonstrate successful alteration of a single
base pair in the beta-globin gene from normal to sickle.
NaPro is now investigating the use of the same approach to correct mutant sickle
cells to a normal state with the ultimate hope of providing effective therapy
for sickle cell disease.
"The study results are a big step forward. We are excited to make progress in
our strategy to apply our gene editing technology to our initial focus area of
treatment -- hematologically-based hereditary diseases," stated Jeffrey White,
President of NaPro's Genomics Division.
About the Study
With this study, NaPro accomplished its initial proof of concept, which was to
produce a sickle mutation in normal human hematopoietic stem/progenitor cells
and to detect the production of sickle hemoglobin. Data from the study include
the detection nine weeks post-treatment of edited blood-derived cells, and
importantly, the evidence of altered hemoglobin in red blood cells derived from
treated stem/progenitor cells.
The objective of the study was to develop a procedure using synthetic modified
oligonucleotides to produce gene correction in human hematopoietic
stem/progenitor cells, as well as to quantify the resulting production of
altered hemoglobin.
About Sickle Cell Disease
Sickle cell disease affects approximately 150 million people worldwide, and in
the U.S. approximately one in five hundred African-Americans and one in one
thousand Hispanic-Americans. The disease is characterized by debilitating pain,
severe infections, chronic anemia, and over time, organ damage. It is estimated
that the cost to the U.S. healthcare system for a single sickle patient is in
excess of $500,000 over his or her lifetime. The life expectancy is 42 and 48
years respectively for men and women with this disease.
About NaPro BioTherapeutics, Inc.
NaPro BioTherapeutics, Inc., is a life science company focused in two distinct
areas: the development and in-licensing of targeted anti-cancer agents and the
development of genomic therapies, primarily through the use of "gene editing"
with additional applications in diagnostics, agribiotechnology, and
pharmacogenomics.
The statements in this news release that are not historical facts are
forward-looking statements that represent management's beliefs and assumptions
as of the date of this news release, based on currently available information.
Such forward-looking statements include statements regarding the potential of
the gene editing technology and the success of any clinical applications of the
gene editing technology to sickle cell disease. Forward-looking statements can
be identified by the use of words such as "believes," "intends," "estimates,"
"may," "will," "should," "anticipated," "expected" or comparable terminology or
by discussions of strategy. Although the Company believes that the expectations
reflected in such forward-looking statements are reasonable, it cannot assure
that these expectations will prove to be correct. Such statements involve risks
and uncertainties including, but not limited to, risks associated with
development of the gene editing and sickle cell technologies and businesses,
including other competitors in the genomics and sickle cell disease fields,
delays with or non-approval from regulatory authorities, limitations on the
ability to market products because of the intellectual property rights of third
parties, the ability to obtain, maintain, and enforce patents, and the risk that
development of the sickle cell disease therapy will require new investment.
Should one or more of these risks materialize, or should the underlying
assumptions prove incorrect, actual results could differ materially from those
forecasted or expected. These factors are more fully described in the Company's
documents filed from time to time with the Securities and Exchange Commission,
which are incorporated by reference, including the Company's Annual Report on
Form 10-K and 10-K/A for the year ending December 31, 2002 filed with the
Securities and Exchange Commission on March 27 and April 30, 2003, and the
Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange
Commission on May 16, 2003. The Company disclaims any intention or obligation to
update publicly or revise such statements whether as a result of new
information, future events, or otherwise.
SOURCE NaPro BioTherapeutics, Inc.
ARIAD Reports Method for Treating Eye Diseases That Cause Blindness - Similar to Arresting Angiogenesis in Cancer Study Receives Excellence-in-Research Award by American Society of Gene Therapy
CAMBRIDGE, Mass., Jun 5, 2003 (BUSINESS WIRE) -- ARIAD Pharmaceuticals, Inc.
(Nasdaq: ARIA) today announced, for the first time, results of long-term studies
in non-human primates on a new method that may be used to treat eye diseases
which are the leading causes of vision loss in the Western world. Nearly 3
million patients in the United States are affected by age-related macular
degeneration and diabetic retinopathy; both diseases are characterized by
unwanted new blood-vessel formation - a process known as angiogenesis. This
research highlights a novel treatment method based on ARIAD's cell-signaling
technology that may be used to block angiogenesis locally in the eye using a
targeted and carefully regulated dosing regimen - a method that is similar to
inhibiting the blood supply recruited by tumors.
The study by Lebherz et al from the University of Pennsylvania and ARIAD was
recognized by an Excellence-in-Research Award from the American Society of Gene
Therapy (ASGT) and will be presented at the Society's annual meeting that begins
today.
"This potential new treatment method for two of the most debilitating forms of
eye disease is a direct extension of research in the cancer field where
inhibition of angiogenesis has been shown to be an effective therapy for solid
tumors such as colon cancer," said Harvey J. Berger, M.D., chairman and chief
executive officer of ARIAD. "This research may create partnership and funding
opportunities outside of our core area of product development - cancer
therapeutics."
Eleven studies demonstrate the versatility and safety of the Company's
ARGENT(TM) cell-signaling regulation technology are being presented at the ASGT
meeting. The presentations illustrate the potential clinical use of ARIAD's
technology in various conditions, including type-1 diabetes, lysosomal storage
diseases, anemia and genetic diseases. Two reports also show the safety of
long-term use of the ARGENT system in large animal models, including the absence
of any signs of leukemia or other forms of cancer that have been associated with
certain types of gene therapy.
ARIAD's cell-signaling regulation technology is now being used by over 650
academic investigators worldwide in diverse areas of research. More than 170
papers have been published describing its broad applicability, including during
the past year the creation of clinically relevant animal models of human
disease, such as congestive heart failure and hepatitis. Such animal models may
be invaluable in accelerating the development of new drugs for these diseases.
In January of this year, GPC Biotech AG became the first company to enter into a
commercial R&D license for ARIAD's cell-signaling regulation technology. GPC
Biotech is using ARIAD's technology both in its internal drug discovery efforts
and in partnership with pharmaceutical and biotechnology companies and has
agreed to make payments to ARIAD related to both uses.
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